Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1

What are the differences in clinical outcomes in children with spinal muscular atrophy (SMA) type 1 receiving first-line gene therapy vs nusinersen?In this comparative effectiveness study of 24 children in 12 matched pairs using data from the French National SMA Registry, children treated with first-line gene therapy experienced lower incidence of unsatisfactory clinical response and required less respiratory and nutritional support over time compared with those treated with first-line nusinersen, with similar motor outcomes.The findings support consideration of gene therapy as a potentially preferable treatment option in SMA type 1 and may offer evidence to guide treatment decisions.

Corresponding Author: Juliette Ropars, MD, PhD, Reference Center for Neuromuscular Disorders, Paediatric Department, University Hospital, 2 Avenue Foch, 29609 Brest, France (juliette.ropars@chu-brest.fr).

Link to the article in your story

We encourage you to link out to this article in your story using the link below. It includes an access token that will give free access to the article for your readers up to one year after publication. (The link will be live after the article publishes and embargo is lifted.)

Please see the article for additional information, including full author list, author contributions and affiliations, conflict of interest and financial disclosures, and funding and support.

Need more information? Contact us.