Tag: SMA

Neurology

SUNFISH: Risdiplam Meets Primary Endpoint in SMA

Newsemia
Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in...
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Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newsemia
James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on...

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