Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels ...

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

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

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

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

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 ...