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Nature Medicine|August 9, 2024
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorderAlban Ziegler, Joanne Carroll, Jennifer M Bain, et al.Journal of Neuromuscular Diseases|February 20, 2025
Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophyRafael Rodriguez-Torres, Cara H Kanner, Emma L Gay, et al.Pageof 2