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…
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Over the next 20 years, the number of Americans with spinal muscular atrophy (SMA) will rise from 10,000 to nearly…
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for…
It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact,…
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed…
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: spinal muscular…
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes…
When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress —…
In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning…
At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency…
Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period…
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