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A new phase 1b study by Biogen is now evaluating the drug salanersen in patients with SMA who have already received the gene therapy Zolgensma.
On December 18, 2024, the FDA approved Ryoncil (Remestemcel-L-rknd) as the first and only off-the-shelf allogeneic bone marrow-derived mesenchymal stromal cell therapy for children who are two months old to adolescents with steroid-refractory acute graft-versus-host disease (SR-aGVHD).
Metabolic health has been a hot topic for the last couple of years, and that trend looks to continue in 2026.
