The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).

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 ...

UBS analyst Matthew Weston maintained a Buy rating on Roche Holding AG (RHHVF – Research Report) today and set a price target of CHF338.00. The ...

PTC Therapeutics (NASDAQ:PTCT – Get Free Report) is projected to post its quarterly earnings results after the market closes on Thursday, February 27th. Analysts expect PTC Therapeutics to post ...