Cookies help us deliver our services. By using our services, you agree to our use of cookies. Learn more

News Links

Title Category News Date Link
This Gainsville mom wants to raise awareness of rare disease with upcoming walk at Wilshire Trail article 2021/08/17
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington article 2018/10/03
'It Will Consume Your Life': 4 Families Take On Rare Diseases article 2020/07/11
'Revolutionary' DNA test can detect thousands of rare diseases in children article 2020/01/03
10 Rare Autoimmune Diseases That Don’t Get Enough Attention article 2020/08/15
11 rare skin diseases article 2020/12/27
13 Things ER Staff Should Know About People With Rare Diseases article 2019/08/25
13-Year-Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/10/04
15-month-old Denver girl fighting rare disease, raising awareness for treatments and a cure article 2019/01/05
25-year-old faces rare form of ALS that took her twin, but experimental drug offers hope article 2019/06/22
27 Years of Humanitarian Programs in Vietnam article 2019/12/22
4 deeply personal stories about the impact of genetic testing article 2020/01/26
5 Questions: Stephen Montgomery on RNA’s role in diagnosing rare diseases article 2019/06/22
5 Things You Should Know About Stiff-Person Syndrome article 2020/05/30
5 Things You Should Know About Vasculitis article 2020/03/21
7 Pro Tips to Help Deal With the Anxiety of Rare Disease Parenting article 2019/12/28
7 rare diseases a rural Wisconsin doctor found in his Amish and Mennonite patients article 2019/10/19
7,000 challenges: The basis and burden of rare diseases article 2021/02/28
8-year-old Camryn Kellam Beating Back Blood Disease, Rare Brain Syndrome video 2021/08/22
8-Year-Old Girl Is Left Paralyzed After COVID Likely Triggered Rare Disease article 2021/09/19
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived article 2020/08/02
A father on the legacy of his son's ultra-rare disease podcast 2021/08/29
A Genetic Test For A Microscopic Problem Came With A Jumbo Price Tag article 2020/04/05
A Harvard geneticist is creating a dating app that matches users based on DNA, and people are worried it's eugenics article 2019/12/15
A Helpful Online Safety Guide for People With Autism Spectrum Disorders article 2020/07/01
A new treatment for rare muscular disease article 2021/04/17
A patient-powered registry boosts the study of rare Castleman disease article 2021/01/31
A race against time: Young boy and his family cope with rare disease article 2020/09/12
A Rare Human Disease Has Been Found in Dinosaur Bones, Could it Lead to a Treatment? article 2020/02/19
A rare-disease entrepreneur follows an unconventional drug development path article 2018/10/19
A Xi'an girl's escape from early death sheds light on China's arduous journey to make drugs affordable for rare diseases article 2019/07/09
A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving podcast 2020/06/27
A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment podcast 2019/12/28
Access And Actionability Are Key For Genetic Testing And Precision Medicine article 2020/01/18
Actors Who Have Rare Diseases And Medical Conditions article 2021/10/03
Acute Necrotizing Encephalopathy: Moree community supports two-year-old Paige Thomas as she fights rare brain disease article 2018/08/21
Addressing Diagnosis Challenges in the Rare Disease Space article 2018/10/19
Adult Polyglucosan Body Disease: Film series pick highlights rare disease - Jewish Exponent article 2018/07/25
Advances in inborn errors of metabolism article 2019/01/26
Aeglea BioTherapeutics Receives FDA Rare Pediatric Disease Designation For ACN00177 For The Treatment Of Homocystinuria article 2020/12/06
After Timesia Hart Was Diagnosed With a Rare Disease, She Started a Bike Race to Inspire Others article 2021/07/07
AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu article 2018/07/26
AI Rapidly Diagnoses Rare Disorders in Critically Ill Children article 2021/10/16
AI technology can identify genetic diseases by looking at your face, study says article 2019/01/15
Alexion and Affibody Announce Partnership to Co-Develop Anti-FcRn Affibody® Molecule article 2019/03/25
Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria article 2018/08/01
Alnylam's Givosiran Looks Positive Overall in Phase III Trial for Rare Disease article 2019/03/07
Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington article 2018/07/29
An experimental test may help confirm cases of chronic fatigue syndrome article 2019/05/02
An experimental test may help confirm cases of chronic fatigue syndrome article 2019/05/08
Angelman Advocate Launches ‘Combined Brain’ Rare Disease Consortium article 2019/12/12
Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia article 2018/07/21
Antioch teen with rare disease won’t let COVID-19 stop her article 2020/03/21
Apic Bio’s APB-102 Receives Orphan Drug Designation from the FDA for the Treatment of Genetic SOD1 ALS article 2019/07/23
Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant article 2018/08/17
Apple to Participate in Meeting Advocating for Better Patient Access to Health Info article 2020/01/26
Army major walking barefoot from Cornwall to Edinburgh to fund treatment for daughter with rare disease article 2020/07/11
AstraZeneca drug for rare disease shows promise in flushing copper build-up article 2021/08/29
Autosomal Dominant Alzheimer's Disease: Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/09/25
Axovant Licenses Two Gene Therapies Aimed at Tay-Sachs and Sandhoff Disease article 2018/12/19
Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes article 2016/07/07
Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes article 2016/06/27
Barefoot major finishes 700 mile walk for daughter article 2020/08/15
Batten Disease: Norfolk Tides help Virginia Beach teen with rare disease article 2018/08/21
Benefit held for local woman battling rare disease video 2018/11/18
Biallelic HMBS Deficiency: 'Kure 4 Kam': Parents navigate medical maze so 1-year-old son might withstand rare disease article 2018/08/24
BioMarin stands tall with first approved drug for rare disease that causes dwarfism article 2021/08/29
Boehringer Ingelheim Gambles $2.62 Billion on Two Fibrotic Disease Deals article 2019/07/23
Boy's Severe Headaches Lead to Rare Eye Disease Diagnosis article 2019/09/01
Boy, three, who was diagnosed with a rare neurological condition which affects just one in a BILLION, is able to walk for the first time after his parents raised £16k for a 'life-changing' machine article 2019/08/25
BridgeBio Pharma And Affiliate Origin Biosciences Announces FDA Acceptance Of Its New Drug Application For Fosdenopterin For The Treatment Of MoCD Type A article 2020/10/11
British father begins 1,200- mile barefoot walk in Maine to raise awareness for daughter's rare disorder video 2021/08/29
CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo article 2018/07/16
California group helps people with rare diseases aim for new heights article 2018/08/16
California legislature poised to establish Rare Disease Advisory Council article 2021/09/19
California parents hope for miracle after baby diagnosed with rare, fatal genetic disorder article 2019/05/19
Canadians Invited To Share Their Views On A National Strategy For High-Cost Drugs For Rare Diseases article 2021/01/31
CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com article 2018/07/12
CDER’s Progress in Rare Diseases article 2021/02/28
CDISC and NORD Partner to Develop Data Standards for Rare Diseases article 2021/04/24
Cecilia woman testifies to Congress about rare disease article 2021/07/31
CENTOGENE Discovers Six New Rare Diseases by Leveraging the Strength of Its Bio/Databank article 2021/05/09
Chan Zuckerberg Initiative launches new program to support rare disease patients article 2019/06/16
Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases article 2020/02/09
Change the genes to fix the skin article 2019/04/04
Cheers, tears as Staten Island teen with rare disease receives new home article 2018/11/28
ChemoCentryx's drug gets U.S. FDA nod for treating rare autoimmune disease article 2021/10/10
Chiesi creates Boston rare disease unit ahead of Fabry launch article 2020/02/09
Chiesi Global Rare Diseases Announces FDA Approval of FERRIPROX® (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease article 2021/05/02
Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for Pegunigalsidase Alfa for Proposed Treatment of Fabry Disease video 2021/01/16
Chiesi Global Rare Diseases Recognizes Fabry Disease Awareness Month and Launches “Fabry Focus on Health” Educational Series to Support Patients and Caregivers During COVID-19 Pandemic article 2020/04/25
Child fights for her life by taking the 'most expensive drug in the world' video 2021/05/21
China is picking up the fight against rare diseases article 2019/06/30
Chronic Lyme disease is a rare, but serious condition — here's how to know if you have it article 2020/08/24
CMN: 'For someone to see the way I look as art is amazing' article 2019/03/18
Coalition Will Address Racial Disparities in Rare Disease Communities article 2020/06/14
Columbus mom lost one daughter to a rare disease. A new drug is helping another video 2021/06/12
Commentary: What those with rare diseases want us to consider article 2020/05/05
Commercialization Imperatives for Success in Rare Diseases article 2019/09/15
Community Hero: Grant Bonebrake champions those with rare diseases and wins national award article 2020/12/21
Community rallies around 6-year-old with rare disease. His family needs your help. article 2021/07/11
Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com podcast 2018/07/19
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds article 2021/04/03
Could CRISPR Be the Key for Rapidly Aging Children? article 2021/01/10
Could gene therapy cure sickle cell anemia? video 2019/03/11
Could This Radical New Approach to Alzheimer’s Lead to a Breakthrough? article 2019/02/10
COVID Vaccine for Patient With Rare Disease? Best Guess Is Yes article 2021/01/24
COVID's all-hands-on-deck approach should be standard for rare diseases article 2021/03/21
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say article 2020/05/30
COVID-19 in a patient with systemic sclerosis treated with tocilizumab for SSc-ILD article 2020/04/02
COVID-19 pandemic hinders access to care for patients with rare diseases article 2020/05/10
Customized gene therapies successfully target rare eye diseases article 2020/09/12
Cystic Fibrosis article 2020/11/15
Cystic Fibrosis: One Patient’s Story, Current Treatments and Exciting New Therapies on the Horizon article 2021/05/16
Cystinosis: Firelight Fund to help local family as child battles rare disease article 2018/09/11
CZI awards $1.3 million to support the global rare disease community article 2020/07/19
Dansville teen overcomes rare disease, returns to dance article 2018/12/09
Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan article 2018/07/24
Denali's Hunter Syndrome Candidate Gets Orphan Drug Status article 2019/06/16
Denver family fundraising to help toddler suffering from rare disease video 2019/08/05
Designer Microbes: a Potential Living Cure for Crohn's and Phenylketonuria article 2019/04/21
Detecting rare disease: Revealing the methods, motivations, and implications article 2021/04/17
Devastated mum loses all three kids to rare disease found in just 110 people in UK article 2021/02/21
Developing RNA Medicines for Rare Genetic Diseases article 2018/09/11
Diagnoses of rare diseases enhanced through the teamwork of national network article 2020/11/01
Doctors said boy wouldn't live past his second birthday. His town just threw a parade for his third article 2019/11/24
Dr. Google' Helps Some Patients Diagnose a Rare Disease article 2019/09/01
Dravet Syndrome: Community rallies around 3-year old battling rare disorder article 2018/08/25
Drew Brees gives emotional speech as Steve Gleason receives Congressional Gold Medal article 2020/01/18
Drug Discovery for Rare Diseases article 2019/06/22
Drug offers hope for patients with primary hyperoxaluria type 1 article 2020/06/08
Drug shortage threatens health of O'Fallon man video 2019/07/09
Dubai Science Park Raises Focus on Rare Diseases During Expert Panel article 2018/09/30
Dublin Boy With Rare Disease Gets Special Bike article 2018/11/28
Dublin teen's film on rare disease chosen for international festival article 2020/04/12
Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat article 2018/07/19
Ducks’ Brady Breeze ‘repping for rare diseases’ at pro day video 2021/04/03
Dying patients with rare diseases struggle to get experimental therapies video 2021/07/11
Early study points to potential therapeutic avenue for a pair of rare pediatric diseases article 2021/02/13
Early Study Shows Promise of CRISPR Injection to Treat Rare Disease article 2021/07/18
East Bay family launches national campaign to save life of 5-year-old who needs bone marrow transplant article 2019/01/20
Eddie Vedder Leads Star-Studded Event To Raise Awareness And Funds For Rare Disease article 2020/11/15
Ehlers-Danlos Syndrome: Madera boy with rare disease using YouTube to raise awareness article 2018/09/06
Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies article 2020/03/29
Emedgene collaborates with Illumina to scale the interpretation of genomic data for rare diseases article 2020/12/21
Enrollment in the U.S. Phase 2 Clinical Trial in Cystic Fibrosis Has Resumed After Being Paused in Response to the COVID-19 Pandemic article 2020/08/15
Epidermodysplasia Verruciformis: New Link Found Between A Rare Skin Disease and Beta-HPV article 2018/08/16
Epidermolysis Bullosa: 'My skin tears and blisters at the slightest touch, but it won't stop me from modelling' article 2018/08/30
ERN-LUNG Project Helping People With Rare Lung Diseases article 2021/01/31
Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun article 2018/07/29
EU support for Italian biotech's red blood cell technology to treat rare diseases article 2020/08/08
European Commission approves nintedanib for the treatment of systemic sclerosis-associated interstitial lung disease (SSc-ILD) article 2020/04/25
European initiative targets diagnosis, treatment of rare diseases article 2019/08/05
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients article 2019/06/22
Evans syndrome: Everything you need to know article 2020/02/23
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US article 2020/08/24
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US article 2021/04/17
Exclusions: Adding Complexity for Patients with Rare Diseases article 2018/10/10
Exclusions: Adding Complexity for Patients With Rare Diseases article 2018/12/25
Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/10/05
Experimental Therapy Gives Children With Genetic Disorders A New Chance At Life video 2019/03/25
Fabry Disease: FDA Approves Galafold for Rare Genetic Disorder, Fabry Disease article 2018/08/14
Familial Amyloid Polyneuropathy: What caused this man's scalloped pupil? article 2018/07/31
Familial Chylomicronemia Syndrome: Ultra-rare disease patients find support in connectedness article 2018/08/21
Fanconi Anemia: Jimbo Fisher spreads awareness of son's blood disease through nonprofit article 2018/08/31
FDA Aims to Ease Single-Patient Trials for Rare Disease Drugs (1) article 2021/01/31
FDA Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC article 2018/07/29
FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL article 2020/07/29
FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease article 2020/04/12
FDA Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease article 2020/06/16
FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder article 2019/05/12
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels article 2019/09/29
FDA Approves First Treatment For Hutchinson-Gilford Progeria Syndrome And Some Progeroid Laminopathies article 2020/11/22
FDA approves first treatment for inherited rare disease article 2019/12/12
FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A article 2021/02/28
FDA approves first treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system article 2019/06/30
FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder article 2021/06/05
FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease article 2018/11/28
FDA approves first treatment to increase pain-free light exposure in patients with a rare disorder article 2019/10/13
FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease article 2020/08/04
FDA Approves New Therapy for Dravet Syndrome article 2020/06/25
FDA Approves New Therapy for Myelodysplastic Syndromes (MDS) That Can Be Taken at Home article 2020/07/07
FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord article 2020/06/14
FDA approves new treatment for rare disease of the bone marrow and blood article 2019/01/05
FDA Approves Oral Treatment for Spinal Muscular Atrophy article 2020/08/07
FDA Approves Orphan Drug Pemigatinib for Rare Bile Duct Cancer Cholangiocarcinoma article 2020/06/10
FDA approves pexidartinib for tenosynovial giant cell tumor article 2019/08/02
FDA Approves REBLOZYL® (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions article 2019/11/17
FDA approves treatment for patients with rare bone marrow disorder article 2019/08/17
FDA Approves Treatment for Rare Disease Affecting Optic Nerves, Spinal Cord article 2020/08/24
FDA Awarding Grants for Research into Rare Diseases article 2018/09/25
FDA Awarding Grants for Research into Rare Diseases article 2018/09/26
FDA awards 11 clinical trial grants for rare diseases, many in children article 2021/10/16
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases article 2019/10/13
FDA Grants Rare Pediatric Disease Designation to AmideBio’s Treatment for Congenital Hyperinsulinism article 2020/08/24
FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis article 2020/09/27
FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development article 2020/01/18
FDA is Working to Bridge Gaps and Meet Needs for Rare Disease Product Development article 2019/03/18
FDA poised to approve $5 MILLION gene therapy to treat muscular atrophy - making it the most expensive drug on the planet article 2019/05/19
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases article 2019/02/02
FDA's Banner Year For Approvals: Will Market Access To New Drugs Follow Suit? article 2019/01/26
Finding Answers: Statesville woman's rare disease needs rare treatment article 2019/01/05
Finding New Uses For Old Drugs - A Lifeline For Rare Disease Patients article 2019/03/01
First Signs of Huntington's Disease Detectable Via a New Blood Test article 2018/10/17
First US attempt to cure a rare disease with genome editing fails miserably article 2019/02/10
Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org article 2018/03/07
Flying the flag for research in Aarskog syndrome article 2020/10/11
For two UB scientists, love means studying daughter’s rare disease article 2021/08/08
Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech article 2018/08/19
Fulcrum Therapeutics Presents Updated Data On Sickle Cell Disease Program At The 62nd American Society Of Hematology (ASH) Annual Meeting And Exposition article 2020/12/06
Gap in care, research and funding for rare blood disorder disproportionately affecting Black Americans video 2021/05/09
GBT Snags FDA Approval for First-of-its-Kind Sickle Cell Disease Treatment article 2019/11/27
Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports article 2020/06/10
Gene therapy targets sickle-cell disease article 2019/04/04
Gene therapy to halt rare form of sight loss article 2020/02/17
Genethon: green light from the ANSM to start a gene therapy trial for Duchenne Dystrophy article 2020/12/06
Genetic screening for newborns yields some answers, more questions article 2019/01/05
Genomics in healthcare: How systems are using the data and whether there is a cause for concern article 2020/03/12
Getting a Treatment Is Not the End of the Road in Rare Diseases article 2020/09/06
Girl with Uncombable Hair Syndrome is finally able to have her hair brushed after nine years article 2021/01/10
Global Liver Institute Announces First Annual Rare Liver Diseases Month article 2021/02/08
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19 article 2020/03/21
Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland article 2018/07/12
Guest column: Innovative testing detects rare diseases in critically ill infants article 2021/02/21
Guillain-Barre Syndrome: What we know about the disease Travis Frederick is fighting article 2018/08/25
Guillain–Barré Syndrome Associated with SARS-CoV-2 article 2020/04/25
hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease article 2018/08/10
Have 12 friends? You probably know someone with a rare disease article 2019/08/11
He's the oldest-known man with rare Proteus syndrome and he hopes to help find a cure article 2020/02/02
Healx Pushing New Drug Development Model in Rare Diseases with Barth Syndrome Foundation article 2019/03/01
Healx: AI-driven drug repurposing for rare disease article 2021/07/24
Hearing loss and rare diseases: What's the connection? article 2019/05/19
Helping Patients With Rare Dieases During the Coronavirus Pandemic video 2020/04/12
Hereditary Angioedema: Shire wins FDA approval for potential blockbuster rare disease drug article 2018/08/24
Hermansky-Pudlak Syndrome: MN man battles rare genetic disorder article 2018/07/31
How a Collaborative Ecosystem is Driving the Development of Successful Therapies in Rare Disease Research article 2018/09/11
How an ultra-rare disease is bringing this N.S. family closer together article 2021/01/03
How artificial intelligence can help detect rare diseases article 2019/06/16
How Can I Best Prepare for an ABA Therapist in My Home? article 2020/08/11
How Can We Improve the Diagnosis and Treatment of Rare Diseases? article 2020/03/15
How coronavirus affects young adults with chronic illnesses and rare diseases article 2020/07/26
How Covid-19 is changing rare diseases research article 2021/03/13
How Covid-19 is changing rare diseases research article 2021/03/21
How FDA's Breakthrough Therapy Designation Program Changed the Rare Disease Space article 2018/10/28
How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025 article 2018/11/18
How people in China with rare diseases afford outrageously expensive medicines? article 2020/11/09
How People With Weakened Immune Systems Are Navigating The Coronavirus podcast 2020/03/21
How the human genome transformed study of rare diseases article 2021/02/13
How the rare disease community has developed fertile ground for progress article 2020/11/29
How to Rethink Population Health Management for Rare Disease Care article 2020/12/27
How UK startup Mendelian hopes to use machine learning to diagnose rare diseases article 2019/04/20
Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana article 2018/07/17
Hunter Syndrome: Talking about rare disease article 2018/08/13
Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report article 2018/07/10
Hurler Syndrome: World record attempt aims to help local boy battle rare disease article 2018/08/21
I don't know of anyone else': Man with rare disease pens book to help others video 2021/01/10
I have a rare disease. Research on such illnesses can provide insights into more common ones. article 2019/02/19
I have a rare disease. This is my hope for the future of medicine article 2019/05/19
I ran from my lethal diagnosis for years. Let's make this rare disease a household word. article 2020/10/11
I want to cure my son of his rare genetic disease. Is that wrong? article 2020/06/21
ICER discriminates against people with rare diseases article 2019/10/05
Identifying and recruiting patients into rare disease clinical trials: A multi-pronged and customized approach article 2020/11/01
Immune Thrombocytopenic Purpura in a Patient with Covid-19 article 2020/04/30
In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder podcast 2019/07/30
In developing new drugs for rare diseases, this non-profit steps in when pharmaceutical companies won't article 2019/01/15
In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical Relief Program article 2020/05/17
Infographic: Which Rare Diseases Are The Most Common? article 2019/09/22
Inspirational boy battling rare disease returns from hospital after 'life-saving' surgery article 2020/04/19
Internet can be valuable tool for people with undiagnosed rare disorders article 2019/08/11
Is Artificial Intelligence The Best Solution For Diagnosing Rare Diseases? article 2021/07/18
Is technology the key to accelerating rare disease clinical trials? article 2021/10/16
Islanders embrace families whose children have rare diseases article 2021/02/13
Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk article 2018/08/14
KC family worries cost could keep drug from helping son with rare disorder video 2021/06/12
Keenan: Reseachers are 'in this together' to solve rare diseases article 2021/04/10
Krystal Biotech and GeneDx Announce Collaboration to Provide No-charge Genetic Testing for Patients with Suspected Dystrophic Epidermolysis Bullosa (DEB) article 2021/10/16
Kubota Vision Receives Orphan Products Clinical Trials Grants to Emixustat for Stargardt Disease article 2020/08/24
L1 Cam Syndrome: $1K Needed To Help Dublin Boy With Rare Disease In Need Of Bike article 2018/08/20
Lack of treatment options for rare diseases prompts call for change article 2018/09/10
Lamellar Ichthyosis: Mum says her baby girl’s skin is like ‘petting a SNAKE’ due to rare condition that means she has to bathe in bleach article 2018/08/02
Las Vegas woman writes children's book to help people with rare disease video 2021/05/21
Legislation to Help Diagnose Children with Rare Diseases Introduced by Senators Collins, Kelly, and Menendez article 2021/06/27
Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease article 2018/08/15
Lewisville family helps to raise awareness for rare disease article 2020/07/01
Life for a child with a rare disease is a grueling roller coaster ride: A mother explains article 2019/03/06
Live bacteria deliver crucial enzymes straight to the gut article 2018/12/22
Local family shares their journey battling rare disease article 2018/10/19
Local Hero with rare disease to be honored at 2018 MG Walk at Bear Creek Pioneers Park article 2018/11/18
Local nonprofit helping patients with ultra-rare diseases article 2020/12/13
Lorain County family devoted to patient advocacy with rare disease foundation article 2020/10/03
Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A) article 2019/02/19
Lysosomal Acid Lipase Deficiency Financial Assistance Program Launched by The Assistance Fund article 2019/01/26
M6P Therapeutics Receives Six Rare Pediatric Disease Designations from the U.S. FDA for Company’s Deep Pipeline of Programs for Lysosomal Storage Disorders article 2021/01/31
Man diagnosed with rare disease takes 2 life-changing steps article 2018/12/09
Med student uses 'Zebra Alphabet' to bring awareness to rare diseases article 2018/09/03
Medical Detectives: The Last Hope for Families Coping with Rare Diseases article 2018/12/19
Medical Detectives: The Last Hope For Families Coping With Rare Diseases article 2019/01/05
Medical foods are vital to treat rare diseases — but some insurers won't pay article 2018/09/25
Medicine by machine: Is A.I. the cure for the world’s ailing drug industry? article 2020/02/09
Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research article 2018/12/09
Meet the family working with scientists to fight rare diseases with CRISPR therapy video 2020/06/21
Meet the man who invented Viagra: He's fighting rare diseases with artificial intelligence to treat patients video 2020/02/09
Mercer Island family raises awareness for rare, undiagnosed diseases article 2020/02/23
Metopirone Safely Normalized, or Significantly Lowered, Urine Cortisol Levels in Phase 3/4 Trial article 2020/09/19
Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease article 2020/09/19
Mindfulness training shows promise for people with MS article 2020/05/26
Mirum Snags First Drug Approval for Rare, Genetic Liver Disorder article 2021/10/03
Mitochondrial Disease: 13 Year Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/09/25
MMS support program to help ultra-rare disease research article 2021/09/19
Moderna donates ultra-rare disease therapy to nonprofit founded by late Takeda R&D chief article 2021/09/11
Moderna to develop mRNA therapeutic for ultra-rare disease article 2021/09/25
Mom Blazes Path to Cure for Son’s Rare Disease article 2019/03/06
Mom Is Tirelessly Raising Awareness About Her Daughter's Rare, Life-Threatening Disease article 2019/04/20
Moms search for lifesaving bone marrow donors for daughters with rare diseases article 2021/05/16
Montreal doctors’ 'Hail Mary’ treatment could save babies' lives from most common of all rare diseases article 2019/05/08
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist article 2019/03/18
More than a quarter of rare disease trials are culled due to low patient rates: report article 2021/03/13
Most dollars spent on top-selling orphan drugs don’t go to treat people with rare diseases article 2021/03/06
Most ER Staff Don't Understand Rare Disease, So This Is How I Advocate for Myself article 2019/11/24
Moyamoya: The rare disease you've never heard of video 2019/01/20
MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte article 2018/07/18
My Constant Insomnia Turned Out to Be a Symptom of a Rare Disorder Called Hypophosphatasia article 2020/07/29
My friend chose an assisted death in Switzerland. Her dying wish was to tell you why article 2020/06/21
My life with face blindness article 2019/09/01
National Gathering of the Rare Disease Community and Health Professionals Launched with 2019 Living Rare, Living Stronger | NORD Patient & Family Forum article 2018/12/09
Nature Outlook: Huntington's Disease article 2018/10/28
Nature Outlook: Huntington's Disease article 2018/10/28
Nature Outlook: RNA Therapies article 2020/02/02
Navigating the Rare Disease Space: Insights from a Danish CEO article 2018/12/19
New Data-sharing Program Aims to Speed Innovation in Rare Diseases article 2021/10/03
New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many article 2021/09/11
New Hope for Rare Mitochondrial Diseases: An Interview With Minovia Therapeutics’ Natalie Yivgi Ohana article 2019/07/23
New osteosarcoma treatment receives rare pediatric disease designation from the FDA article 2020/10/03
New stem cell source offers hope to patients with rare liver disease article 2021/10/16
New Streaming Channel Showcases Rare Disease Films article 2020/08/15
New Study Proves the Internet Can Help You Get a Rare Disease Diagnosis article 2019/08/20
New taxonomy of non-skeletal rare disorders with impact on bone article 2021/01/16
Newborn Screening Practices and Alpha-Thalassemia Detection — United States, 2016 article 2020/09/12
Newer methods may boost gene therapy's use for more diseases article 2021/06/05
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases article 2018/09/10
NGLY1 Mutation: A Battle Plan for a War on Rare Diseases article 2018/09/10
Niemann-Pick Disease: Reno twins with rare, fatal disease will be on CNN show "Something's Killing Me" article 2018/08/17
NIH funding bolsters rare diseases research collaborations article 2019/10/05
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research article 2019/03/06
NIH study suggests people with rare diseases face significantly higher health care costs article 2021/10/24
NIH-funded study finds gene therapy may restore missing enzyme in rare disease article 2021/07/18
NIH-supported research survey to examine impact of COVID-19 on rare diseases community article 2020/05/10
No Deal Brexit could endanger lives of four million Brits with rare diseases article 2020/12/13
NORD and Partners Host FDA, CDC Leaders in Webinar Addressing Rare Disease Community on COVID-19 Vaccines article 2021/01/31
NORD Launches Financial Assistance Program for Rare Disease Community Members Impacted by COVID-19 article 2020/04/25
North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte article 2018/07/24
Once free, drug for rare disease now being sold for $375K a year video 2019/02/11
One in 67 Hongkongers suffers from a rare disease, study finds article 2018/09/25
One in 67 Hongkongers suffers from a rare disease, study finds article 2018/10/03
Opinion: Genome sequencing can lead to life-changing care for infants. California should make it more available article 2021/04/10
Opinion: Value-based agreements could disrupt how we pay for new therapies article 2019/04/12
Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results article 2021/02/13
PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights article 2019/05/08
Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial article 2018/10/16
Pandemic Won’t Stop Rare Disease Day on Feb. 28 article 2021/02/13
Parents create nonprofit to find cure for toddler's rare disease video 2021/08/22
Partners in crime: Genetic collaborator may influence severity of the rare disease, NGLY1 article 2021/01/31
Patients: The Catalysts Behind Rare Disease Research & Drug Development article 2018/10/28
Peer support groups help mum of child with a rare liver disease find answers and support article 2020/08/24
People with rare autoimmune diseases at increased risk of dying during COVID-19 pandemic article 2020/12/06
People With Rare Diseases Need Better Social Support article 2020/10/18
Pfizer Steps Further In The Door For Gene Therapies article 2019/03/25
Photographs reveal people behind rare illnesses article 2021/07/11
Podcast Extra: The quest for a rare disease treatment podcast 2019/01/15
PODCAST: Rare In Common: Hanging Out With Zebras: Life with a genetic mutation so rare it has never been reported before - RareInCommon.com podcast 2018/02/28
PODCAST: Rare Unplugged: Episode 1 podcast 2018/07/30
Potential Gene Therapy Safely Restores FVIII Clotting Factor in Animals and Human Cells, ASC Therapeutics Reports article 2019/05/08
Potential treatment for rare degenerative disease discovered article 2020/08/02
Priscilla Chan talks her new 'Rare As One' initiative to combat rare diseases video 2019/09/09
ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP) article 2019/08/17
ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa article 2019/12/12
Providing new hope for children suffering from rare muscle diseases article 2020/10/11
Push to spur more drugs for deadly rare diseases article 2021/03/07
Raising awareness for rare diseases matters every day article 2021/03/07
Raleigh biopharma company hopes to have a new treatment for celiac disease article 2021/06/05
Rallying Toward a Better Future for Pompe Disease article 2020/09/12
Randall Rutta: Discrimination against people with rare diseases article 2020/01/26
Rare Artist 2020 award recipients webpage 2021/01/31
Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines article 2021/06/20
Rare congenital malformation caused by epigenetic mechanism in previously mysterious genome sequences article 2021/02/13
Rare disease acromegaly finds an Ottawa champion article 2018/11/18
Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest article 2020/08/08
Rare Disease Companies Unite to Advocate for Life-Changing Therapies article 2021/05/16
Rare Disease Diversity Coalition Awards $600K to Combat Disparities article 2021/10/16
Rare Disease Doctors Are Struggling. Here's 3 Ways Patients and Families Can Help. article 2020/05/24
Rare disease drove two women on a mission to change FDA drug review process article 2020/07/19
Rare Disease Q&A: What Rare Diseases Are and Why That Matters article 2019/12/15
Rare Disease R&D Investments Likely To Grow In Biopharma article 2019/05/02
Rare Disease R&D Investments Likely To Grow In Biopharma article 2019/05/08
Rare Disease Spotlight: Sarcoidosis article 2021/09/25
Rare Disease Therapy Development and Access Remain Top FDA Priorities During COVID-19 article 2020/06/11
Rare Disease Trials Require Patient Insights And Input article 2018/10/16
Rare Diseases 2021: running rare disease trials post-Covid article 2021/09/25
Rare diseases are not uncommon, and may be the mother lode for the world's pharmaceutical producers and researchers article 2019/07/09
Rare diseases are not uncommon, and may be the mother lode for the world’s pharmaceutical producers and researchers article 2019/08/05
Rare Diseases in Asia-Pacific article 2020/08/02
Rare diseases in China, their high treatment costs, and the people publicising patients’ plight article 2019/04/12
Rare diseases in the Bronze Age article 2021/03/06
Rare diseases more common than we think article 2019/11/10
Rare diseases prompted care in ancient times article 2019/07/28
Rare diseases require radical new funding methods article 2018/11/18
Rare diseases the next target for mRNA therapies article 2021/05/16
Rare Diseases: A Psychiatric Concern? article 2019/04/04
Rare Diseases: My teeth fall off easily and my bones are so hard walking breaks them article 2020/08/30
Rare disorder offers roadmap for understanding roots of inflammatory disease article 2021/07/31
Rare eye condition was behind da Vinci's genius article 2018/10/21
Rare Gut Condition A Model For Study Of Genetic Diseases article 2019/04/12
Rare Illness in Children May Be Related to Coronavirus, European Doctors Are Warned article 2020/05/05
Rare Neurological Disease Treatment Market to Record CAGR of 8.7% Increase in Revenue by 2026 article 2020/02/09
Rare Pediatric Disease Designation Granted to Stargardt Disease Treatment, LBS-008 article 2018/09/30
Rare Youth Project: Can you empower the next generation of rare disease advocates? article 2018/11/28
Ravenna boy battles rare disease article 2018/11/18
RDMD Lands $14M to Expand Reach of Rare Disease Research Platform article 2020/04/19
Reauthorize the Creating Hope Act to help kids with rare diseases article 2020/09/27
Recordati Rare Diseases Inc. Announces Availability of ISTURISA® (osilodrostat) in the United States article 2020/06/14
RECORDATI RARE DISEASES: CARBAGLU® (Carglumic Acid) Tablets 200mg Receives U.S. FDA Approval for a New Indication to Treat Acute Hyperammonemia Associated with Propionic Acidemia and Methylmalonic Acidemia article 2021/01/31
Report: Orphan Drug Act Boosted Rare Disease Therapies, But Access is Uneven article 2021/03/27
Researchers create an animal model for studying GPI anchor deficiencies article 2021/01/10
Researchers develop language test for people with Fragile X syndrome article 2020/06/01
Researchers develop new therapeutic approach to treat CLN3 Batten disease article 2020/08/02
Researchers find a potential new combination therapy against a rare disease article 2019/01/05
Researchers lead national efforts around childhood blood disorders article 2020/10/18
Researchers use patients’ cells to test gene therapy for rare eye disease article 2021/01/31
Retrophin buys up Orphan Technologies, spending $90M on rare disease drug article 2020/10/24
Rich and Caitlin Hill pledge $575,000 to MGH for genetic disease research article 2019/03/01
Richard Engel Celebrates Son's 5th Birthday, Shares Message to All Kids with Rett Condition article 2020/10/03
Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD article 2020/10/11
Russia’s Coronavirus Outbreak Jeopardizes Care for Patients With Rare Diseases article 2020/04/25
Same But Different Photo Contest Celebrates People With Rare Diseases article 2020/09/12
Sangamo Announces 16 Week Clinical Results Including Reductions In Glycosaminoglycans In Phase 1/2 Trial Evaluating SB-913, A Zinc Finger Nuclease Genome Editing Treatment For MPS II (Hunter Syndrome) article 2018/12/25
Sanofi Sees Positive Results in Two Clinical Trials for Niemann-Pick Disease article 2020/02/02
Sanofi Snags FDA Approval for Rare Blood Disorder article 2019/02/10
Sarcoidosis: Local nurse leads support group for rare disease article 2018/08/05
Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease article 2018/10/28
Scientists discover new genetic disease that delays brain development in children article 2021/04/03
Scientists suggest new solution to the rare-disease problem article 2019/12/15
September, 2009 | Patient Voices: Rare Diseases - The New York Times article 2009/09/01
She was asked to speak about her two ‘ultrarare’ diseases. She did, while also telling of a rare place. article 2020/03/04
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide article 2020/04/19
Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide article 2020/05/10
Skeletal Rare Diseases Academy launched by IOF article 2020/09/19
Sobi and Sanofi Donate up to 500 Million Additional IUs of Clotting Factor to WFH Humanitarian Aid Program article 2020/06/14
Software engineer bets on technology to help speed rare disease treatments article 2021/04/03
Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others article 2018/08/27
St. Jude gene therapy cures babies with 'bubble boy' disease article 2019/04/20
Stars who suffer from rare diseases article 2021/02/28
STAT Act Offers Hope to People Living with Rare Diseases article 2021/03/21
Stiff Skin Syndrome: 'I email 100 doctors a day': Mother of boy, 12, with rare terminal disease that is turning him 'to STONE' describes her desperate attempts to find a cure article 2018/07/30
Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People article 2018/07/18
Stranger's bone marrow donation saves 1-year-old baby girl with rare 'bubble boy' disease article 2019/07/13
Students transform school into Hogwarts to surprise classmate fighting rare disease article 2018/11/18
Study Pins Down Number of Americans with Most Common Form of Lupus article 2021/01/31
Study provides new hope for children suffering from rare muscle diseases article 2020/10/18
Study reveals biochemical alterations in patients with Lesch-Nyhan disease article 2020/06/10
Summerside boy with rare disease gets his wish to visit Legoland in Florida article 2018/10/19
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe article 2020/12/13
Taysha Gene Therapies Receives Rare Pediatric Disease And Orphan Drug Designations For TSHA-105 For The Treatment Of Epilepsy Caused By SLC13A5 Deficiency article 2021/01/24
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say article 2020/11/09
Testing for rare diseases at birth may have spared our girl a life of suffering, says mother of five-year-old who has degenerative brain condition article 2020/03/04
Texas Baby Born Without Skin Leaves Doctors Scrambling for Answers and Battling to Save His Life article 2019/04/20
Thank You for Your COVID-19 Response — from a Rare Disease Patient article 2020/03/28
The Bad Economics of the U.S. Health Care System Shows Up Starkly in Its Approach to Rare Diseases article 2020/03/15
The Challenges Of Combating Rare Diseases - And Five Innovations Making A Real Difference article 2020/03/04
The Champion for Rare Disease Cures article 2021/09/19
The Conundrum Of Investing In Cardiovascular Versus Rare Diseases R&D article 2020/01/26
The coronavirus pandemic claims another victim: Medical research for deadly rare diseases article 2020/06/07
The Importance of Research on Rare Diseases article 2020/03/15
The Loneliness I Feel as the Parent of a Child With a Rare Disease article 2021/06/20
The needs of disabled children are being overlooked in the wake of covid-19 article 2020/09/19
The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price article 2021/06/05
The poor diagnosis of rare diseases: Overcoming deficits in information, awareness, and understanding article 2021/03/27
The rare condition slowly paralysing my arms and legs article 2020/12/06
The rare disease challenge meeting article 2019/02/19
The UK Rare Diseases Framework article 2021/01/16
These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them. article 2019/02/02
These two Raytown women have a rare disease. Why don’t more doctors want to help? video 2019/08/11
They don't know if their children will ever walk or talk. But finding other families online has given them hope. article 2019/05/12
This college dropout was bedridden for 11 years. Then he invented a surgery and cured himself article 2019/07/28
This med student was given last rites before finding a treatment that saved his life. His method could help millions article 2019/09/15
This Penn Professor Almost Died From a Rare Disease Four Times article 2019/09/01
Top 5 Most-Read Rare Disease Stories of 2020 article 2021/01/03
Toronto family of child with ultra-rare disease urges public to take COVID-19 seriously article 2020/03/28
Toronto parents of baby needing $2.8M drug turn to Swiss drugmaker's dose lottery as a last resort article 2020/01/01
Treating Polycythemia Vera article 2021/05/16
Treatments for rare diseases at risk, due to the decline of plasma donations article 2020/12/13
Two Patient Deaths Halt Audentes’ Gene Therapy Trial article 2020/07/02
U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease article 2019/05/08
U.S. patients with rare diseases go to the doctor an average of 8 times to find answers video 2019/11/05
Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases article 2021/02/21
Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome article 2019/08/17
Understanding genetics of common, rare disorders advance treatment in pediatric neurology article 2020/09/19
Undiagnosed rare diseases costing NHS over £3bn, study shows article 2018/12/19
Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest article 2018/08/02
Unprecedented data sharing driving new rare disease diagnoses in Europe article 2021/06/05
UPCOMING EVENT: The Amyloidosis Foundation's Annual 'Run for Your Life' webpage 2018/10/13
UPCOMING VIRTUAL EVENT: The 2018 Rare Fair webpage 2018/09/28
Updated Study Analyzes Use and Cost of Orphan Drugs article 2018/10/28
Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness article 2021/09/25
Using DNA sequencing data with electronic health records to find rare variants behind inherited diseases article 2021/01/16
Using telehealth to revolutionize the speed of making rare disease diagnoses article 2020/08/30
Vaccines and rare diseases: using one to help the other article 2020/10/24
VIDEO: Rare In Common video 2017/02/27
VIDEO: Super Power Baby Project - TEDxAuckland video 2014/10/09
View from inside: Rare diseases in the times of COVID19 article 2020/12/13
Virtual gala to raise funds, awareness for rare neurodevelopmental disorder article 2021/05/02
Vivek Ramaswamy's Latest "Vant": Aruvant to Focus on Sickle Cell and Beta-Thalassemia article 2018/11/28
Voices from the front line in rare disease article 2020/08/02
Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco article 2018/07/12
Website tackles an all-too-common problem with rare diseases: A shortage of information article 2019/12/15
What I’ve Learned Since My Diagnosis with a Rare Blood Cancer article 2019/04/04
What My Family and Friends Want You to Know About Mal de Debarquement Syndrome article 2020/07/01
What to Do If Your Child Is Diagnosed With a Rare Disease article 2020/03/21
What to Do When You’re Diagnosed with a Rare Disease article 2018/10/01
What to know about paroxysmal nocturnal hemoglobinuria (PNH) article 2021/10/16
What you need to know about HLH, the rare disease linked to ESPN reporter Edward Aschoff's death article 2020/01/13
When a Rare Mutation Causes a Rare Disease: Jacob’s Story article 2019/06/22
When a Rare Mutation Causes a Rare Disease: Jacob’s Story article 2019/06/22
When I Developed the Disease My Father Was Researching article 2019/06/02
When I Had No Choice But to Go to the Hospital During COVID-19 for My Rare Disease article 2020/05/24
When Your Child With a Rare Disease Doesn’t Feel Brave article 2019/10/28
Who's Equipped to Deal With Pandemic Worries? Patients With Rare Diseases, Some Say article 2020/04/19
Why I Hate My Kid's Birthday article 2021/05/16
Why We Decided to Start the First Rare Disease Film Festival article 2019/10/28
With $3m in funding, tech firm looks to drive drug development using EHR data article 2018/08/21
With a nudge from AI, ketamine emerges as a potential rare disease treatment article 2021/08/08
Woman hits the road to support Rare Disease Cause video 2021/08/22
Woman with rare disease delivers healthy baby article 2020/10/11
Woman with rare muscular disease sings to exercise her lungs. Her songs inspire others to chase their dreams video 2021/05/02
Women with a Rare Disease Take Control of Their Destiny article 2018/11/18
Yeast Models Provide New Insights into Neurodegenerative Diseases article 2021/10/03
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases article 2019/09/01
‘I Just Want My Kid To Have A Good Life’: Colorado Boy 1 In 40 Worldwide Diagnosed With Rare Disease video 2021/07/31
‘My immune system is very susceptible’: Woman with rare disease wants you to take COVID-19 seriously video 2020/03/15
‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases article 2021/08/29
‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access article 2021/05/21
‘You can carry on or give up’: Families living with rare diseases article 2021/03/07
£1m step closer to understanding genetic diseases article 2020/12/13