Last Updated: 09/26/2026
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Found 5 publications
Safety, Tolerability, and Efficacy of a Prophylactic Sirolimus Protocol for Patients Receiving Delandistrogene Moxeparvovec-Rokl Gene Therapy.
Journal: Human gene therapy
Published: August 05, 2026
Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN-Digenic Myopathy.
Journal: Annals of clinical and translational neurology
Published: February 04, 2026
New SMA era: A broad-range tiered assessment of function for the evolving SMA phenotype (EVOLVE-SMA).
Journal: Journal of neuromuscular diseases
Published: December 30, 2025
Spinal Muscular Atrophy-Survivorship and Care in a New Therapeutic Landscape.
Journal: Pediatric neurology
Published: July 21, 2025
Top 10 Clinical Pearls in Inherited Neuropathies.
Journal: Seminars in neurology
Published: October 15, 2024
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Last Updated: 09/26/2026