Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

Leader of Opposition Suvendu Adhikari donated Rs 10 lakh from his salary towards the treatment of a girl from West Bengal's ...

A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...

PTC Therapeutics, Inc. (NASDAQ:PTCT – Get Free Report)’s share price dropped 8.8% during trading on Monday after JPMorgan ...

Survey finds that 59 per cent of those planning a family have concerns about the slow pace of newborn screening expansion ...

Research analysts at Redburn Atlantic began coverage on shares of Ionis Pharmaceuticals (NASDAQ:IONS – Get Free Report) in a ...

Dr. Spela Mirosevic signs a historic €1 million funding agreement with the Slovenian government - the first public investment in nonprofit-led gen ...

Learn more about whether Corcept Therapeutics Incorporated or Novartis AG is a better investment based on AAII's A+ Investor ...

Free Ben & Jerry’s cones   Ben & Jerry’s annual Free Cone Day is Tuesday, April 8, from Noon – 8PM. The Ben […] ...

Columnist Kevin Schaefer has found that living with SMA has meant its share of sitcom scenarios, some with an underlying ...