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News Links

Title Category News Date Link
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington article 2018/10/03
13-Year-Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/10/04
A rare-disease entrepreneur follows an unconventional drug development path article 2018/10/19
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
AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever - LABIOTECH.eu article 2018/07/26
Alkaptonuria: New treatment for ultra-rare disease, alkaptonuria article 2018/08/01
Alternating Hemiplegia of Childhood: DC Lemonade Stand Raises More Than $1K for Toddler With Rare Disease - NBC Washington article 2018/07/29
Angelman Syndrome: Angelman Syndrome: The rare condition stopping kids from walking and talking - nine.com Australia article 2018/07/21
Aplastic Anemia: Boise teen with rare disease finds one in a million match for life-saving transplant article 2018/08/17
Autosomal Dominant Alzheimer's Disease: Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/09/25
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
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
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
CD40 Ligand: "Baby" King is not a baby anymore: Syracuse boy with rare disease ha outlived the odds - Syracuse.com article 2018/07/12
Cheers, tears as Staten Island teen with rare disease receives new home article 2018/11/28
Congenital Thrombotic Thrombocytopenic Purpura: SU football's Sam Heckel fights a rare disease; Boeheim's army is back - Syracuse.com podcast 2018/07/19
Cystinosis: Firelight Fund to help local family as child battles rare disease article 2018/09/11
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
Developing RNA Medicines for Rare Genetic Diseases article 2018/09/11
Dravet Syndrome: Community rallies around 3-year old battling rare disorder article 2018/08/25
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
Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat article 2018/07/19
Ehlers-Danlos Syndrome: Madera boy with rare disease using YouTube to raise awareness article 2018/09/06
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
Erythropoietic Protoporphyria: Genetic condition makes toddler allergic to sun article 2018/07/29
Exclusions: Adding Complexity for Patients with Rare Diseases article 2018/10/10
Exercise may slow cognitive decline in people with rare Alzheimer's disease article 2018/10/05
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 Announces New Framework for Gene Therapies to Treat Rare Diseases - AJMC article 2018/07/29
FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease article 2018/11/28
FDA Awarding Grants for Research into Rare Diseases article 2018/09/25
FDA Awarding Grants for Research into Rare Diseases article 2018/09/26
First Signs of Huntington's Disease Detectable Via a New Blood Test article 2018/10/17
Five Things I've Learned About Living With a Rare Disease - GlobalGenes.org article 2018/03/07
Frontotemporal Dementia: St. George support group focuses on rare disease that causes loss of speech article 2018/08/19
Glycogen Storage Disease: Cleveland teen helps to research her own disease - WKYC Cleveland article 2018/07/12
Guillain-Barre Syndrome: What we know about the disease Travis Frederick is fighting article 2018/08/25
hATTR: FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease article 2018/08/10
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 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
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
Kawasaki Disease: Rare disease leaves Cambridgeshire toddler unable to walk and talk article 2018/08/14
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
Leigh Syndrome: A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease article 2018/08/15
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
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 foods are vital to treat rare diseases — but some insurers won't pay article 2018/09/25
Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research article 2018/12/09
Mitochondrial Disease: 13 Year Old Battles Rare Disease That Leaves Him Unable to Eat Solid Food article 2018/09/25
MPS 7: Baby battling rare disease arrives home after receiving potentially lifesaving treatment - WSOCTV Charlotte article 2018/07/18
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
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
North Carolina to require more screening for rare diseases in newborns - WSOC Charlotte article 2018/07/24
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
Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial article 2018/10/16
Patients: The Catalysts Behind Rare Disease Research & Drug Development article 2018/10/28
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
Rare disease acromegaly finds an Ottawa champion article 2018/11/18
Rare Disease Trials Require Patient Insights And Input article 2018/10/16
Rare diseases require radical new funding methods article 2018/11/18
Rare eye condition was behind da Vinci's genius article 2018/10/21
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
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
September, 2009 | Patient Voices: Rare Diseases - The New York Times article 2009/09/01
Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others article 2018/08/27
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
Students transform school into Hogwarts to surprise classmate fighting rare disease article 2018/11/18
Summerside boy with rare disease gets his wish to visit Legoland in Florida article 2018/10/19
Undiagnosed: Picture of Ontario girl with rare disease wins Ontario Youth Sports photo contest article 2018/08/02
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
VIDEO: Rare In Common video 2017/02/27
VIDEO: Super Power Baby Project - TEDxAuckland video 2014/10/09
Vivek Ramaswamy's Latest "Vant": Aruvant to Focus on Sickle Cell and Beta-Thalassemia article 2018/11/28
Waardenburg Syndrome: If One Eye’s Brown and One Eye’s Blue, This Day is for You - KWTX Waco article 2018/07/12
What to Do When You’re Diagnosed with a Rare Disease article 2018/10/01
With $3m in funding, tech firm looks to drive drug development using EHR data article 2018/08/21
Women with a Rare Disease Take Control of Their Destiny article 2018/11/18