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No findings reported in abstract; leaves open all clinical and research questions.
M.F. is a site principal investigator for Roche and Novartis Gene Therapies Inc. SMA clinical trials, and the institution has received funds for contract research related to the conduct of these trials.
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Mandarakas et al. (2024) studied Spinal Muscular Atrophy. Patient-Reported Outcomes was evaluated.
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