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New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? L CARNITINE increases muscle mass, reduces fat mass. Bangkok Hospital Headquarter Advances in therapy for spinal muscular atrophy: promises and challenges Nature Reviews Neurology 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 SMA CARNI VAL TRIAL PART II: A Prospective, Single Armed Trial of L Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy PLOS One
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