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

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.

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 ...

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

A retrospective analysis on newborn screening for spinal muscular atrophy (SMA) provides further evidence for the benefits of ...

Scholar Rock stock skyrockets as the late-stage study of the investigational therapy, apitegromab, to treat spinal muscular atrophy meets the primary goal.

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

The biotech company Scholar Rock reported that its experimental medicine succeeded in a late-stage trial of patients with ...

NDAQ:SRRK) Scholar Rock Reports Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular ...

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