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Spinal muscular atrophy (SMA) is a neuromuscular disease caused by low levels of SMN protein. Several therapeutic approaches boosting SMN are approved for human patients, delivering remarkable improvements in lifespan and symptoms. However, emerging phenotypes, including neurodevelopmental comorbidities, are being reported in some treated SMA patients, indicative of alterations in brain development.
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Division of Nephrology, Boston University Chobanian & Avedisian School of Medicine, Boston, United States of America.
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Rac1 palmitoylation is required for cardiac stress adaptation and regulation of Protein Kinase A signaling.
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