Duchenne muscular dystrophy future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Fahimeh Shojaei, M.D.

Overview

Future or investigational therapies for Duchenne muscular dystrophy include gene therapy, creatine monohydrate, myostatin inactivation, cell therapy, and idebenone (antioxidant).

Future or investigational therapies for Duchenne muscular dystrophy include:^[1]^[2]^[3]^[4]^[5]

Gene therapy
- Injecting microdystrophin genes through recombinant adeno-associated viral (rAAV) vectors
Creatine monohydrate
- Increases muscle strength
Myostatin inactivation
- Increase muscle mass and strength
Cell therapy
- Isolating and transplanting muscle satellite cells as a skeletal muscle progenitor.
Idebenone (antioxidant)

↑ Konieczny P, Swiderski K, Chamberlain JS (May 2013). "Gene and cell-mediated therapies for muscular dystrophy". Muscle Nerve. 47 (5): 649–63. doi:10.1002/mus.23738. PMC 4077844. PMID 23553671.
↑ Walter MC, Lochmüller H, Reilich P, Klopstock T, Huber R, Hartard M, Hennig M, Pongratz D, Müller-Felber W (May 2000). "Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study". Neurology. 54 (9): 1848–50. PMID 10802796.
↑ Bogdanovich S, Krag TO, Barton ER, Morris LD, Whittemore LA, Ahima RS, Khurana TS (November 2002). "Functional improvement of dystrophic muscle by myostatin blockade". Nature. 420 (6914): 418–21. doi:10.1038/nature01154. PMID 12459784.
↑ Cerletti M, Jurga S, Witczak CA, Hirshman MF, Shadrach JL, Goodyear LJ, Wagers AJ (July 2008). "Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles". Cell. 134 (1): 37–47. doi:10.1016/j.cell.2008.05.049. PMC 3665268. PMID 18614009.
↑ Buyse GM, Voit T, Schara U, Straathof C, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T (May 2015). "Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial". Lancet. 385 (9979): 1748–1757. doi:10.1016/S0140-6736(15)60025-3. PMID 25907158. Vancouver style error: initials (help)