The FDA approved Itvisma, the first gene replacement therapy for children 2 years and older, teens, and adults with spinal muscular atrophy, a one-time treatment that replaces the SMN1 gene and demonstrated improved motor function in Phase 3 studies.
Experts counter Robert F. Kennedy Jr.’s claims about aluminum adjuvant safety, noting extensive evidence that aluminum-containing vaccines remain effective and well-validated.
Arrowhead’s small interfering RNA therapy reduced triglycerides and lowered pancreatitis risk in the phase 3 PALISADE trial, providing a quarterly self-administered option for patients with familial chylomicronemia syndrome.