Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a ...

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The health activists argue that the government is empowered with effective law and policy tools and therefore should ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

Filmmaker Reid Davenport weaves a powerful first-person perspective with a journalistic investigation about disability ...

Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.

We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...

SPINRAZA is an approved treatment for spinal muscular atrophy in over 71 countries ... of the higher dose regimen in children and adults who transitioned from SPINRAZA 12 mg to the 50/28 mg ...