With Spinraza Available, Cure SMA Chief Urges Nationwide Screening for Disease
Over the next 20 years, the number of Americans with spinal muscular atrophy (SMA) will rise from 10,000 to nearly…
Articles by Larry Luxner
#ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for…
Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases
It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact,…
NIH Agency Pioneers Collaborative Research into Rare Diseases
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed…
Connecticut Neurologist Upbeat on New Therapies for SMA, Other Disorders
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: spinal muscular…
Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes…
‘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 —…
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…