FDA approves 1st-of-its-kind treatment for rare muscle disease

The FDA has approved Scholar Rock’s ISEMBYLD for spinal muscular atrophy in adults and children ages 2 and older who are currently receiving a survival motor neuron 2-targeted treatment. ISEMBYLD is the first muscle-targeted treatment approved for children and adults with SMA who are receiving an SMN2-targeted treatment. The approval was based on results from […] The post FDA approves 1st-of-its-kind treatment for rare muscle disease appeared first on Becker's Hospital Review |...