Roche’s Evrysdi (risdiplam) tablets have been approved by the European Commission (EC) to treat spinal muscular atrophy (SMA) ...

Panelists discuss how disease progression despite treatment in older patients with spinal muscular atrophy (SMA) may be due to natural aging effects combined with SMA, not necessarily treatment ...

Scholar Rock , a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (SMA) and other severe and debilitating ...

Physiotherapy, is essential for cancer patients to improve physical function, quality of life, and overall health.

Screening can now determine their risk for an ever-growing list of conditions — including ones we can’t do much about.

Casma Therapeutics, Inc., a biotechnology company engaging the autophagy and lysosomal systems to develop innovative new medicines, today announced the nomination of its first development candidate, ...

Artificial intelligence is the latest technology stoking fears of human decline. Early studies stoke doubt about whether the ...

RVPH Open Label Extension Key Opinion Leader Event Reviva Pharmaceuticals Holdings, Inc. (NASDAQ:RVPH) held a key opinion ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

XMEA is a recessive, genetic disease that causes progressive muscle weakness. Can a small fish help identify possible ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...