A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...

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

The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...

Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for ...

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 ...

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...

Evrysdi is currently being, or has been, evaluated in numerous global multicentre trials in people with SMA: FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants ...

Basel, 12 February 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam ...