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FDA approves Scholar Rock’s ISEMBYLD for spinal muscular atrophy patients aged 2 and older on SMN2 therapy

PUBT·09/11/2026 22:25:19
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FDA approves Scholar Rock’s ISEMBYLD for spinal muscular atrophy patients aged 2 and older on SMN2 therapy
  • FDA cleared Scholar Rock’s ISEMBYLD (apitegromab-mstn) for spinal muscular atrophy in adults, children aged 2+ on SMN2-targeted therapy.
  • Phase 3 SAPPHIRE showed a 2.2-point HFMSE gain versus placebo at one year; 34.2% achieved ≥3-point improvement.
  • U.S. launch underway; product expected to ship in coming days.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Scholar Rock Holding Corporation published the original content used to generate this news brief via Business Wire (Ref. ID: 202609111825BIZWIRE_USPR_____20260911_BW564629) on September 11, 2026, and is solely responsible for the information contained therein.