FDA approves Isembyld to treat muscle loss in spinal muscular atrophy patients.
The FDA has approved Isembyld, a new drug to treat muscle loss in patients with spinal muscular atrophy (SMA). The drug is approved as an add-on to existing therapies for the national, rare disease population of people aged 2 and older. The approval was based on clinical trial improvements in motor skills, and long-term real-world outcomes are not yet known.
Why it matters
This provides progress for spinal muscular atrophy patients by offering the first approved therapy that specifically targets muscle loss, a problem not fully addressed by existing treatments.
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