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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A new movement in medicine aims to teach “shame competence,” helping physicians recognize and manage one of the field’s most corrosive yet overlooked emotions.
Bavarian Nordic’s freeze-dried Jynneos vaccine offers logistical advantages over the liquid-frozen version, supporting long-term U.S. stockpiling and public health preparedness.