#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
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2018/10/03 |
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13-Year-Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food
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2018/10/04 |
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15-month-old Denver girl fighting rare disease, raising awareness for treatments and a cure
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2019/01/05 |
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A rare-disease entrepreneur follows an unconventional drug development path
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2018/10/19 |
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Acute Necrotizing Encephalopathy: Moree community supports two-year-old Paige Thomas as she fights rare brain disease
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2018/08/21 |
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Addressing Diagnosis Challenges in the Rare Disease Space
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2018/10/19 |
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Adult Polyglucosan Body Disease: Film series pick highlights rare disease - Jewish Exponent
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2018/07/25 |
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Advances in inborn errors of metabolism
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2019/01/26 |
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AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu
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2018/07/26 |
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AI technology can identify genetic diseases by looking at your face, study says
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2019/01/15 |
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Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria
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2018/08/01 |
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Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington
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2018/07/29 |
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Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia
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2018/07/21 |
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Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant
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2018/08/17 |
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Autosomal Dominant Alzheimer's Disease: Exercise may slow cognitive decline in people with rare Alzheimer's disease
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2018/09/25 |
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Axovant Licenses Two Gene Therapies Aimed at Tay-Sachs and Sandhoff Disease
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2018/12/19 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community (Part 2 of 2) - GlobalGenes
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2016/07/07 |
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Banking for the Future: How Biobanking Helps the Rare Disease Community - GlobalGenes
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2016/06/27 |
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Batten Disease: Norfolk Tides help Virginia Beach teen with rare disease
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2018/08/21 |
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Benefit held for local woman battling rare disease
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2018/11/18 |
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Biallelic HMBS Deficiency: 'Kure 4 Kam': Parents navigate medical maze so 1-year-old son might withstand rare disease
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2018/08/24 |
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CADASIL: Family fighting against rare disease through annual fundraiser - KWWL Waterloo
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2018/07/16 |
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California group helps people with rare diseases aim for new heights
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2018/08/16 |
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CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com
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2018/07/12 |
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Cheers, tears as Staten Island teen with rare disease receives new home
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2018/11/28 |
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Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com
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2018/07/19 |
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Could This Radical New Approach to Alzheimer’s Lead to a Breakthrough?
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2019/02/10 |
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Cystinosis: Firelight Fund to help local family as child battles rare disease
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2018/09/11 |
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Dansville teen overcomes rare disease, returns to dance
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2018/12/09 |
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Degenerative Leiomyopathy: Marshall family hopeful they'll find cure for daughter's rare disease - WWMT West Michigan
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2018/07/24 |
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Developing RNA Medicines for Rare Genetic Diseases
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2018/09/11 |
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Dravet Syndrome: Community rallies around 3-year old battling rare disorder
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2018/08/25 |
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Dubai Science Park Raises Focus on Rare Diseases During Expert Panel
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2018/09/30 |
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Dublin Boy With Rare Disease Gets Special Bike
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2018/11/28 |
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Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat
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2018/07/19 |
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East Bay family launches national campaign to save life of 5-year-old who needs bone marrow transplant
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2019/01/20 |
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Ehlers-Danlos Syndrome: Madera boy with rare disease using YouTube to raise awareness
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2018/09/06 |
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Epidermodysplasia Verruciformis: New Link Found Between A Rare Skin Disease and Beta-HPV
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2018/08/16 |
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Epidermolysis Bullosa: 'My skin tears and blisters at the slightest touch, but it won't stop me from modelling'
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2018/08/30 |
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Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun
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2018/07/29 |
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Exclusions: Adding Complexity for Patients with Rare Diseases
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2018/10/10 |
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Exclusions: Adding Complexity for Patients With Rare Diseases
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2018/12/25 |
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Exercise may slow cognitive decline in people with rare Alzheimer's disease
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2018/10/05 |
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Fabry Disease: FDA Approves Galafold for Rare Genetic Disorder, Fabry Disease
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2018/08/14 |
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Familial Amyloid Polyneuropathy: What caused this man's scalloped pupil?
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2018/07/31 |
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Familial Chylomicronemia Syndrome: Ultra-rare disease patients find support in connectedness
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2018/08/21 |
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Fanconi Anemia: Jimbo Fisher spreads awareness of son's blood disease through nonprofit
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2018/08/31 |
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FDA Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC
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2018/07/29 |
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FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease
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2018/11/28 |
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FDA approves new treatment for rare disease of the bone marrow and blood
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2019/01/05 |
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FDA Awarding Grants for Research into Rare Diseases
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2018/09/25 |
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FDA Awarding Grants for Research into Rare Diseases
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2018/09/26 |
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FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
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2019/02/02 |
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FDA's Banner Year For Approvals: Will Market Access To New Drugs Follow Suit?
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2019/01/26 |
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Finding Answers: Statesville woman's rare disease needs rare treatment
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2019/01/05 |
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First Signs of Huntington's Disease Detectable Via a New Blood Test
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2018/10/17 |
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First US attempt to cure a rare disease with genome editing fails miserably
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2019/02/10 |
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Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org
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2018/03/07 |
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Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech
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2018/08/19 |
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Genetic screening for newborns yields some answers, more questions
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2019/01/05 |
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Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland
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2018/07/12 |
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Guillain-Barre Syndrome: What we know about the disease Travis Frederick is fighting
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2018/08/25 |
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hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
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2018/08/10 |
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Hereditary Angioedema: Shire wins FDA approval for potential blockbuster rare disease drug
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2018/08/24 |
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Hermansky-Pudlak Syndrome: MN man battles rare genetic disorder
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2018/07/31 |
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How a Collaborative Ecosystem is Driving the Development of Successful Therapies in Rare Disease Research
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2018/09/11 |
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How FDA's Breakthrough Therapy Designation Program Changed the Rare Disease Space
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2018/10/28 |
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How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025
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2018/11/18 |
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Hunter Syndrome: Bozeman boy’s family looks into gene therapy to cure rare disease - NBC Montana
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2018/07/17 |
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Hunter Syndrome: Talking about rare disease
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2018/08/13 |
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Huntington's Disease: New Therapies on the Horizon for Huntington's Disease - Rare Disease Report
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2018/07/10 |
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Hurler Syndrome: World record attempt aims to help local boy battle rare disease
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2018/08/21 |
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I have a rare disease. Research on such illnesses can provide insights into more common ones.
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2019/02/19 |
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In developing new drugs for rare diseases, this non-profit steps in when pharmaceutical companies won't
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2019/01/15 |
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Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk
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2018/08/14 |
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L1 Cam Syndrome: $1K Needed To Help Dublin Boy With Rare Disease In Need Of Bike
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2018/08/20 |
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Lack of treatment options for rare diseases prompts call for change
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2018/09/10 |
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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
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2018/08/02 |
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Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease
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2018/08/15 |
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Live bacteria deliver crucial enzymes straight to the gut
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2018/12/22 |
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Local family shares their journey battling rare disease
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2018/10/19 |
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Local Hero with rare disease to be honored at 2018 MG Walk at Bear Creek Pioneers Park
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2018/11/18 |
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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)
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2019/02/19 |
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Lysosomal Acid Lipase Deficiency Financial Assistance Program Launched by The Assistance Fund
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2019/01/26 |
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Man diagnosed with rare disease takes 2 life-changing steps
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2018/12/09 |
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Med student uses 'Zebra Alphabet' to bring awareness to rare diseases
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2018/09/03 |
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Medical Detectives: The Last Hope for Families Coping with Rare Diseases
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2018/12/19 |
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Medical Detectives: The Last Hope For Families Coping With Rare Diseases
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2019/01/05 |
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Medical foods are vital to treat rare diseases — but some insurers won't pay
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2018/09/25 |
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Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research
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2018/12/09 |
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Mitochondrial Disease: 13 Year Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food
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2018/09/25 |
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Moyamoya: The rare disease you've never heard of
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2019/01/20 |
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MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte
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2018/07/18 |
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National Gathering of the Rare Disease Community and Health Professionals Launched with 2019 Living Rare, Living Stronger | NORD Patient & Family Forum
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2018/12/09 |
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Nature Outlook: Huntington's Disease
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2018/10/28 |
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Nature Outlook: Huntington's Disease
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2018/10/28 |
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Navigating the Rare Disease Space: Insights from a Danish CEO
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2018/12/19 |
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NGLY1 Mutation: A Battle Plan for a War on Rare Diseases
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2018/09/10 |
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NGLY1 Mutation: A Battle Plan for a War on Rare Diseases
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2018/09/10 |
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Niemann-Pick Disease: Reno twins with rare, fatal disease will be on CNN show "Something's Killing Me"
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2018/08/17 |
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North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte
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2018/07/24 |
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Once free, drug for rare disease now being sold for $375K a year
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2019/02/11 |
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One in 67 Hongkongers suffers from a rare disease, study finds
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2018/09/25 |
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One in 67 Hongkongers suffers from a rare disease, study finds
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2018/10/03 |
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Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial
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2018/10/16 |
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Patients: The Catalysts Behind Rare Disease Research & Drug Development
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2018/10/28 |
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Podcast Extra: The quest for a rare disease treatment
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podcast |
2019/01/15 |
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PODCAST: Rare In Common: Hanging Out With Zebras: Life with a genetic mutation so rare it has never been reported before - RareInCommon.com
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podcast |
2018/02/28 |
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PODCAST: Rare Unplugged: Episode 1
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podcast |
2018/07/30 |
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Rare disease acromegaly finds an Ottawa champion
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2018/11/18 |
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Rare Disease Trials Require Patient Insights And Input
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2018/10/16 |
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Rare diseases require radical new funding methods
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2018/11/18 |
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Rare eye condition was behind da Vinci's genius
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2018/10/21 |
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Rare Pediatric Disease Designation Granted to Stargardt Disease Treatment, LBS-008
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2018/09/30 |
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Rare Youth Project: Can you empower the next generation of rare disease advocates?
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2018/11/28 |
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Ravenna boy battles rare disease
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2018/11/18 |
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Researchers find a potential new combination therapy against a rare disease
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2019/01/05 |
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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)
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2018/12/25 |
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Sanofi Snags FDA Approval for Rare Blood Disorder
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2019/02/10 |
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Sarcoidosis: Local nurse leads support group for rare disease
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2018/08/05 |
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Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease
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2018/10/28 |
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September, 2009 | Patient Voices: Rare Diseases - The New York Times
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2009/09/01 |
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Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others
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2018/08/27 |
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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
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2018/07/30 |
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Stiff Skin Syndrome: 12-year-old's rare disease is turning him to stone - People
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2018/07/18 |
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Students transform school into Hogwarts to surprise classmate fighting rare disease
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2018/11/18 |
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Summerside boy with rare disease gets his wish to visit Legoland in Florida
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2018/10/19 |
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The rare disease challenge meeting
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2019/02/19 |
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These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.
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2019/02/02 |
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Undiagnosed rare diseases costing NHS over £3bn, study shows
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2018/12/19 |
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Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest
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article |
2018/08/02 |
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UPCOMING EVENT: The Amyloidosis Foundation's Annual 'Run for Your Life'
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webpage |
2018/10/13 |
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UPCOMING VIRTUAL EVENT: The 2018 Rare Fair
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webpage |
2018/09/28 |
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Updated Study Analyzes Use and Cost of Orphan Drugs
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2018/10/28 |
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VIDEO: Rare In Common
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video |
2017/02/27 |
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VIDEO: Super Power Baby Project - TEDxAuckland
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video |
2014/10/09 |
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Vivek Ramaswamy's Latest "Vant": Aruvant to Focus on Sickle Cell and Beta-Thalassemia
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article |
2018/11/28 |
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Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco
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2018/07/12 |
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What to Do When You’re Diagnosed with a Rare Disease
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2018/10/01 |
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With $3m in funding, tech firm looks to drive drug development using EHR data
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2018/08/21 |
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Women with a Rare Disease Take Control of Their Destiny
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2018/11/18 |
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