Page 3 - Equitable Access News Today

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.

The Vineyard Montessori School presented around a dozen revisions to its expansion plan during a Martha’s Vineyard Commission (MVC) meeting on Thursday. The revisions include lowering the height of the building’s roof ridge by over two feet, adding a gable roof to the lobby entrance, extending all three upper-level decks for more comfortable entry, modifying […]

Experts delve into the fundamentals of gene therapy, covering in vivo and ex vivo methods, and explore cellular function targets for effective treatment of a disease.

Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression.

Federal agencies should take coordinated action to ensure that data sharing policies incentivize high-quality data management and sharing plans.

Federal grantmakers should establish a default expectation that hardware developed as part of federally supported research be released as open hardware.