Mother took Evrysdi for SMA before birth; baby later received Zolgensma. Dr. Richard Finkel at St. Jude led study showing no ...

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

PTC Therapeutics (NASDAQ:PTCT – Get Free Report) had its price objective hoisted by Royal Bank of Canada from $60.00 to $63.00 in a research report issued to clients and investors on Tuesday,Benzinga ...

While investing in biotech stocks can be risky due to clinical trial failures and regulatory hurdles, successful drug approvals can result in massive stock gains. Let’s look at two such growth stocks ...

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

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.

Test your knowledge of spinal muscular atrophy (SMA) with this quiz covering its genetic causes, diagnosis, and treatment ...

Basel: Roche, announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an ...

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

The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral ...

Novartis NVS and AbbVie ABBV announced small M&A deals. The FDA approved the expanded use of Pfizer’s PFE cancer drug ...