✉news ScienceBiology first seen 5 h ago, last 4 h ago, peak #31
Approved Metabolism Drugs Rescue Disease Signs in SMA Mice
Researchers report that metabolism drugs already approved for other uses reversed signs of spinal muscular atrophy (SMA) in mouse models. Because these treatments have cleared regulatory safety hurdles, the findings could speed up testing in SMA patients. The work is drawing attention in the biology and rare disease communities as a potential new, faster route to therapy beyond existing genetic treatments.
Why now: Repurposing already-approved drugs for a serious neuromuscular disease offers a potentially faster path to human trials
spinal muscular atrophySMA micemetabolism drugs
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Evidence
- Approved Metabolism Drugs Rescue Disease Signs in SMA Mice · bioengineer.org
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