‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event
When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress —…
Articles by Larry Luxner
Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials
In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning…
US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs
At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency…
Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National
Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period…
Bionews to Cover 3 Rare Disease Day Events, Including NIH Conference
In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and…
Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28
A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll…
Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress
David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In…
NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases
This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into…
ALS Association’s Incoming CEO Urges Insurers to Expand Narrow Coverage of Radicava
WASHINGTON — Visitors to the ALS Association’s Washington, D.C., headquarters can’t help but notice the silver baseball bat sitting in…
Cytokinetics Ends Tirasemtiv Program After Potential ALS Therapy Fails at Trial Goals
Expressing “profound disappointment” with tirasemtiv — their lead candidate for amyotrophic lateral sclerosis (ALS) — officials at Cytokinetics…