Med (New York, N.Y.)Muscular DystrophyDuchenneHumansGenetic TherapyOligonucleotides
Antisense and gene therapy trials in Duchenne muscular dystrophy.
Mariacristina Scoto, Francesco Muntoni
Published: 202510.1016/j.medj.2025.100938
Abstract
Duchenne muscular dystrophy (DMD) is severe and the most common form of muscular dystrophy in childhood. There is no cure for this condition, but several approved therapeutic approaches aim to delay disease progression. Multiple ongoing clinical tria…
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