Scholar Rock Gets FDA Approval for Spinal Muscular Atrophy Treatment in Children, Adults; Shares Up Pre-Bell

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Scholar Rock (SRRK) has received US Food and Drug Administration approval for Isembyld to treat spinal muscular atrophy in certain patients aged two years and above, the company said Friday.

Isembyld is expected to ship in the coming days, Scholar Rock said, adding that the company has received a rare pediatric disease priority review voucher that may be used to obtain priority review for a future marketing application.

Spinal muscular atrophy is a severe neuromuscular disease that leads to an irreversible loss of motor neurons and progressive muscle wasting.

Scholar Rock shares were up 8.6% in premarket activity Monday.

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