STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle loss
Summary
The FDA approved Scholar Rock's Isembyld, the first drug designed to directly target muscle loss in spinal muscular atrophy. In a late-stage trial, young patients taking Isembyld alongside an SMN2-targeting treatment showed improved motor skills after one year.
The approval expands SMA treatment beyond correcting the disease's underlying genetic deficit to preserving and
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The drug establishes muscle preservation as a new treatment target in SMA and could reshape care for children who still lose function despite existing therapies.