Because sickle cell disease isn’t as progressive a disease as others that have gene therapies available, there isn’t as much ...

The babies were treated before symptom onset, and are sitting, standing, and walking independently, many consistent with ...

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

Analyst Jason Gerberry of Bank of America Securities maintained a Buy rating on Biohaven Ltd. (BHVN – Research Report), retaining the ...

Scientists compared the CASMA screening approach against standard methods, and found it better at detecting problems in genes ...

Brian Skorney, an analyst from Robert W. Baird, maintained the Buy rating on Biohaven Ltd. (BHVN – Research Report). The associated price ...

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

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

Scholar Rock Holding Corporation (Nasdaq: SRRK), a late-stage biopharmaceutical company focused on advancing innovative ...

Shares of Scholar Rock SRRK skyrocketed 362% on Monday after the company announced positive top-line results from the ...

Scholar Rock aims for a significant market presence with apitegromab in spinal muscular atrophy. See more on SRRK stock and ...