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.
Researchers examine the strengths and shortcomings of the current European Alliance of Associations for Rheumatology definition for difficult-to-treat rheumatoid arthritis.
The FDA’s authorization of the Q-Collar is under scrutiny after researchers identified duplicated data, altered study endpoints, and unsubstantiated “brain protection” claims.