Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202 for the treatment of Duchenne muscular dystrophy will be ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a promising study has found.

Hi friends! Shane here! I just wanted to quick put up some reflections on my day today. I got my 7th Spinraza injection and it was quite a long day!

Biohaven in recent months has reported a clinical stumble in spinal muscular atrophy, alongside a Phase I readout for its ...

In her writ petition, Seba called for the continuation of her treatment with risdiplam beyond the Rs 50 lakh cap under the ...

The U.S. 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). The new tablet is ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

Learn more about whether Ionis Pharmaceuticals, Inc. or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral solution. Today, the FDA approved the first tablet formulation of ...