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Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disease that affects about 1 in every 5,000 boys, and it is the most common fatal X-linked disease in the world. Individuals affected by this disease will start developing muscle weakness by the age of 5, usually lose the ability to walk by their early teens, and ultimately, they will succumb to respiratory or cardiac failure by the age of 30. 

References

  1. Palma CD, Morisi F, Cheli S, Pambianco S, Cappello V, Vezzoli M, Rovere-Querini P, MoggioM, Ripolone M, Francolini M, Sandri M, Clementi E. Autophagy as a new therapeutic target in Duchenne muscular dystrophy. Cell Death and Disease 3, 2012.

  2. Spada ARL. PPARGC1A/PGC-1α, TFEB and enhanced proteostasis in Huntington disease. Autophagy8: 1845–1847, 2012.