Scholar Rock announces new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with apitegromab.
Scholar Rock announces new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with apitegromab.
Data shows that continued treatment with apitegromab over the extended treatment period is associated with substantial and sustained improvement in motor function, as well as improvements in patient-reported outcome measures in patients with nonambulatory Types 2 and 3 spinal muscular atrophy (SMA) receiving survival motor neuron (SMN)-targeted therapy.
The results were presented today, during the Annual Cure SMA Conference, by Thomas Crawford, M.D., of Johns Hopkins Medicine, and the lead principal investigator of the TOPAZ trial.