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.
The FDA’s authorization of the Q-Collar is under scrutiny after researchers identified duplicated data, altered study endpoints, and unsubstantiated “brain protection” claims.
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The FDA approved Bildyos and Bilprevda are providing new options comparable to Prolia and Xgeva for osteoporosis, cancer-related bone disease, and other skeletal conditions.