Excerpt:
The FDA has granted approval to Novartis for Itvisma (onasemnogene abeparvovec-brve), the first gene replacement therapy for spinal muscular atrophy (SMA) available for patients aged 2 years and older. Backed by data from the Phase 3 STEER and Phase 3b STRENGTH trials, this one-time intrathecal treatment addresses the genetic root cause of SMA and is expected to be available in the U.S. this December.
GENETICSNeurologyPHARMACOLOGY < class="penci-entry-title entry-title grid-title penci_grid_title">New Hope for SMA: FDA Approves Itvisma for Ages 2+