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 ...

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

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

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). Evrysdi is the third treatment approved for SMA ...

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

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 ...

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 ...

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 ...