Open accessResearch articleFirst published 2026-03-01pp. 179-189Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy Joel Iff, Isabelle Desguerre, Yunjuan Liu , [...]View all
Open accessReview articleFirst published 2026-03-01pp. 147-155A historical perspective on the development of antisense oligonucleotide treatments for Duchenne muscular dystrophy and spinal muscular atrophy Annemieke Aartsma-RusORCID, Shin'ichi TakedaORCID
Open accessReview articleFirst published 2026-03-01pp. 190-206Real-world evidence on nusinersen treatment of persons with SMA: a focused review Susan E MatesanzORCID, Richard S FinkelORCID
Open accessReview articleFirst published 2026-03-01pp. 167-178Beyond muscle: Delivering RNA therapeutics to the CNS in Duchenne muscular dystrophy Ophélie VaccaORCID, Cathy NagyORCID, Aurélie GoyenvalleORCID