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Duan, D., Goemans, N., Takeda, S. et al. Duchenne muscular dystrophy. Nat Rev Dis Primers 7, 13 (2021). https://doi.org/10.1038/s41572-021-00248-3 Abstract Duchenne muscular dystrophy is…

Smith J, Damm K, Hover G, Chien J. Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease.…

Flotats-Bastardas M, Hahn A. New Therapeutics Options for Pediatric Neuromuscular Disorders. Front Pediatr 2020;8:583877   Abstract Neuromuscular disorders (NMDs) of…

Roy B, Griggs R. Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders. Neurol Clin 2021;39:87-112   Abstract Increased…

Iftikhar M, Frey J, Shohan MJ, Malek S, Mousa SA. Current and emerging therapies for Duchenne muscular dystrophy and spinal…

Wall A, Lee GH, Maldonado J, Magnus D. Genetic disease and intellectual disability as contraindications to transplant listing in the…

Naarding KJ, Doorenweerd N, Koeks Z, Hendriksen RGF, Chotkan KA, Krom YD, et al. Decision-Making And Selection Bias in Four…

Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, et al. Suitability of external controls for drug…

Sun C, Shen L, Zhang Z, Xie X. Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update. Genes. 2020;11(8)   Abstract…

Łoboda A, Dulak J. Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future. Pharmacol Rep 2020…