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Advances in therapy for spinal muscular atrophy: promises and challenges Nature Reviews Neurology Targeting Skeletal Muscle Dysfunction With L Carnitine for the Treatment of Patients With Chronic Obstructive Pulmonary Disease In Vivo Counteracting chromatin effects of a splicing correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy: Cell Spinal muscular atrophy insights and challenges in the treatment era Nature Reviews Neurology
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