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Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease.
Ryan Haumschild, PharmD, MS, MBA, and Mary Pak, MD, FACP, consider FDA-approved gene therapies for hemophilia, spinal muscular atrophy, and Duchenne muscular dystrophy, exploring their genetic foundations, the FDA approval process, and considerations for therapeutic value and cost.
The Vineyard Montessori School presented about a dozen revisions to its expansion plan during a Martha’s Vineyard Commission (MVC) meeting on Feb. 15. The revisions include lowering the height of the building’s roof ridge by more than two feet, adding a gable roof to the lobby entrance, extending all three upper-level decks for more comfortable […]
The University’s course materials transition group is exploring the benefits of a digital coursework equitable access model over the current inclusive access model.
Emma Ciafaloni, MD, FAAN, describes the key differences among viral vectors used in gene therapy, highlighting their applications and strengths in targeting specific diseases.