Scholar Rock Shares Design of Phase 3 SAPPHIRE Trial of Apitegromab

Scholar Rock Shares Design of Phase 3 SAPPHIRE Trial of Apitegromab

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Scholar Rock Shares Design of Phase 3 SAPPHIRE Trial of Apitegromab

Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and young adults with types 2 and 3 SMA who are unable to walk. Apitegromab will be given as an add-on therapy to either Spinraza (nusinersen) or Evrysdi (risdiplam), two SMA disease-modifying therapies (DMTs) approved in the U.S. and Europe. The global trial, called SAPPHIRE, plans to enroll patients across 55…

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