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Molecular Therapy. Methods & Clinical Development|December 2, 2021
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case seriesChristiano R R Alves, Marco Petrillo, Rebecca Spellman, et al.Journal of Neuromuscular Diseases|April 7, 2026
Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMAKristin J Krosschell, Sally Dunaway Young, Elizabeth Maczek, et al.Nature Communications|March 5, 2025
Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1Preeti Kumari, Lauren M Sullivan, Zhaozhi Li, et al.Muscle & Nerve|August 24, 2017
Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type IKristin J Krosschell, John T Kissel, Elise L Townsend, et al.Nature Medicine|August 15, 2025
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trialFlorian Eichler, Oguz I Cataltepe, Rrita Daci, et al.Pageof 3