4.5 Review

The Relationship between Body Composition, Fatty Acid Metabolism and Diet in Spinal Muscular Atrophy

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Summary: The review highlights the challenging journey from gene discovery to therapy in spinal muscular atrophy (SMA), emphasizing the importance of perseverance in uncovering the biological mechanisms of the disease. Despite the impressive improvements seen with three therapeutic strategies in SMA, there are still many unanswered questions that need to be addressed as discussed in the review.

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