Scholar Rock (SRRK), a late-stage biopharmaceutical company, will present additional data from its phase III SAPPHIRE clinical trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19 in Dallas, Texas.
SAPPHIRE is a phase III trial assessing the efficacy and safety of the company's lead drug candidate, Apitegromab, in children aged 2 to 12 with spinal muscular atrophy who are being treated with survival motor neuron (SMN)-targeted therapies, such as Biogen/Ionis' Spinraza or Roche's Evrysdi.
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