The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...
The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
Over the past nine years, patients with spinal muscular atrophy have seen the number of FDA-approved treatment options for ...
New research at the University of Alberta may make it easier to diagnose serious diseases that disrupt the body's energy ...
Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...
Blizzard Entertainment and World of Warcraft players raised over $2 million for CureDuchenne, honoring Mats Steen, a devoted player lost to Duchenne muscular dystrophy, while promoting awareness and ...
Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.
Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.
Cumberland Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug has improved the amount of blood pumped by the heart, ...
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