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- Scholar Rock receives FDA approval for apitegromab (Isembyld) to treat spinal muscular atrophyScholar Rock announced FDA approval for its injectable drug apitegromab, marketed as Isembyld, for treating spinal muscular atrophy, a rare muscle-wasting condition. The approval follows earlier s…
- FDA seeks external input on rare disease trial criteria and endpoint selectionThe FDA announced it will seek additional external feedback as it reviews how to evaluate efficacy and safety data for rare disease therapies. The agency noted the difficulty of setting appropriat…