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

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

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

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

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 ...

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 ...

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

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

StockNews.com lowered shares of PTC Therapeutics (NASDAQ:PTCT – Free Report) from a buy rating to a hold rating in a research note published on Monday morning. Several other analysts have also ...

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more than 44 pounds, according to the manufacturer. RapidAI automated imaging ...

FDA approves the expanded use of PFE and RHHBY's drugs. Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries Evrysdi tablet can be stored at room ...