FDA approves Isembyld, first therapy for muscle loss in spinal muscular atrophy
The FDA approved Scholar Rock's drug Isembyld for US patients with spinal muscular atrophy aged 2 and older who are already on SMN2-targeting treatments. The drug is the first therapy to specifically target muscle loss in this condition. It is approved for use in combination with existing therapies, not as a standalone treatment, and its real-world effectiveness is not yet known.
Why it matters
This approval provides a new treatment option for a specific symptom of spinal muscular atrophy, a disease where existing therapies do not address muscle loss.
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