Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway
SMN-targeted disease-modifying treatments focus on motor neuron survival rather than muscle. Treated individuals nonetheless experience a range of persistent muscle weakness. Treatments that inhibit myostatin signaling represent a potential complementary pathway for direct muscle enhancement. In the evolving SMA treatment landscape, understanding how muscle-targeted treatment can be incorporated into clinical practice will facilitate individualized treatment decisions and identify outcomes that best encapsulate maintenance or improvement of motor function across the phenotypic spectrum of SMA.
Authors
- Mary Schroth (ORCID: https://orcid.org/0000-0002-1720-4849)
- B. Darras
- Thomas E. Brown
- Mouhamed Gueye
- Jena M. Krueger (ORCID: https://orcid.org/0009-0009-3471-3395)
- RS Finkel
- Thomas O. Crawford
- Laurent Servais
Institutions
- St. Jude Children's Research Hospital (US)
- University of Wisconsin–Madison (US)
- Harvard University (US)
- Johns Hopkins University (US)
- Johns Hopkins Medicine (US)
- University of Oxford (GB)
- Scholar Rock (United States) (US)
- Helen DeVos Children's Hospital (US)
- Centre Hospitalier Universitaire de Liège (BE)
Publication Details
- Journal
- Expert Review of Neurotherapeutics
- Published
- 2026-01-22
- DOI
- https://doi.org/10.1080/14737175.2026.2621405
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00