The Paris gold medallist on finding her sport, advocating for wheelchair access and how she and her husband make each other ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

The tablet formulation can either be swallowed whole or dispersed in a teaspoon of filtered (non-chlorinated) water.

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Over the past nine years, patients with spinal muscular atrophy have seen the number of FDA-approved treatment options for ...

The Muscular Dystrophy Association celebrates this new more accessible treatment option for people living with SMA. MDA - New ...

Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and sa ...

Roche (RHHBY) announced that the Food and Drug Administration has approved a New Drug Application for an Evrysdi tablet for people living with ...

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug ...

"The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management." The approval of the Evrysdi tablet was based on the results of a ...

Columnist Helen Baldwin reflects on a quarter century of changes in the SMA community and honors the memory of two ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...