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.

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

A new tablet formulation of SMA treatment Evrysdi (risdiplam) may give patients more "freedom and independence," a Genentech ...

In less than two weeks, the country came together and collected over KGS 510 million (USD 5.9 million), displaying unprecedented unity and generosity.

Gene therapy treatment gives Baby Kiama a lifeline, saving him from world’s most expensive drug that he needed to slow down a ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

The family vehicle helps their teen daughter, who is in a wheelchair, drive more easily. Video from the car caught the act and the suspect’s license plate.

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Neuromuscular diseases encompass a wide range of disorders characterized by progressive muscle weakness and dysfunction, resulting from abnormalities in the ...

Biogen enhances its portfolio and margins amidst market challenges and competition. Click here to find out why BIIB stock is ...

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.