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 ...

Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and safety as the currently ...

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 ...

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 ...

Infants with SMA type 1 born since 2016 experience better functional outcomes and life expectancy with disease-modifying treatments.

A new electrical stimulation therapy for spinal muscle atrophy (SMA) has shown promise in reactivating motor neurons and ...