Tag : Spinal Muscular Atrophy

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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Faster, cheaper, better: improved gene therapy tool production

Newsemia
The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new...
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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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Unique gene therapy approach paves new way to tackle rare, inherited diseases

Newsemia
Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally,...
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Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

Newsemia
A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy using a subcutaneous intrathecal catheter system configured by...

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