The Montreal Children’s Hospital has reported a significant breakthrough: a nine-day-old baby named Samuel has become the youngest patient in North America to receive gene therapy for a rare, degenerative disease called spinal muscular atrophy (SMA). SMA affects...
At the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, Novartis presented new data highlighting the continued efficacy and durability of its gene therapy treatment, Zolgensma, in treating spinal muscular atrophy (SMA). Zolgensma has been shown...
