Researchers have discovered biomarkers for diverse forms of spinal muscular atrophy (SMA) following applications of ...

Two years after treatment, both girls show normal motor function and no signs of spinal muscular atrophy symptoms.

Spinal muscular atrophy (SMA) is a rare genetic disorder that causes muscle weakness and disability, typically in young ...

Majority of children with SMA treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate ...

As his 31st birthday approaches, columnist Kevin Schaefer reflects on how he goes at his own pace while living with SMA.

Scholar Rock stock catapulted by triple digits Monday after its spinal muscular atrophy treatment succeeded in a Phase 3 ...

Scholar Rock contends its experimental drug for spinal muscular atrophy can achieve what none of the currently available therapies can do to treat this rare disease. After positive data from a ...

Phase 3 findings could position the biotech’s therapy to become part of a “new standard” for SMA, its CEO said, while ...

The biotech company Scholar Rock reported Monday that its experimental medicine improved motor function in patients with a muscle-wasting disease called spinal muscular atrophy, paving the way for ...

Precision medicine offers tailored treatments for rare diseases, addressing genetic variations and improving therapeutic ...

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

A phase 3 trial of Scholar Rock’s spinal muscular atrophy (SMA) candidate has hit its primary endpoint, sparking a 200%-plus ...