… It was recently reported that dystrophin is a tumor suppressor gene and likely an anti-… ,
suggesting that therapeutic approaches developed for muscular dystrophies may also serve in the …

… and therapeutics for DMD, but current treatments do not cure the disease. Here, we review
the current status of DMD pathogenesis and therapy, … Discussion and Future Direction …

… molecular-based therapies are introduced. Emerging molecular therapies have evolved in
… Current approaches include novel ASO backbone chemistries with greater stability, 89 as …

… developments in these research areas and specifically focuses on new therapies that are
in … Conclusions and future directions The field of DMD therapy is currently advancing very …

… in establishing the myriad functionalities of dystrophin in striated muscle biology. Here, we
… recent progress toward the development of therapeutic strategies for DMD that are currently …

… approaches in a combined therapy for Muscular Dystrophies. Preclinical studies and clinical
trials revealed that each approach, if used as single therapy, is … and therapeutic treatments …

… line of therapeutic strategies aims to improve muscle function and quality by targeting the
downstream pathological changes, including inflammation, fibrosis, and muscle atrophy. This …

… Future directions Future directions for SMA include gene therapy as a direct way to elevate
SMN protein levels. In SMA mouse models, AAV-mediated SMN1 gene replacement resulted …

… Duchenne muscular dystrophy is a severe, X-linked disease characterized by decreased …
Duchenne muscular dystrophy has allowed for the design of innovative personalized therapies. …

… discusses the current therapies used to treat DMD and explores the therapies currently
being … Duchenne muscular dystrophy is a rare genetic disorder caused by mutations in the X …