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- W2000607588 abstract "Duchenne muscular dystrophy (DMD) is a genetic X-linked recessive orphan disease that affects approximately 1 in 3 500 male births. Boys with DMD have progressive and predictable muscle destruction due to the absence of dystrophin, a protein present under the muscle fiber membrane. This absence induces contraction-related membrane damage and activation of inflammatory necrosis and fibrosis, leading to cardiac/diaphragmatic failure and death. The authors support the therapeutic role of myoblast transplantation in DMD, and describe the history and rationale for such an approach." @default.
- W2000607588 created "2016-06-24" @default.
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- W2000607588 date "2010-09-25" @default.
- W2000607588 modified "2023-09-27" @default.
- W2000607588 title "Myoblast transplantation: A possible surgical treatment for a severe pediatric disease" @default.
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- W2000607588 doi "https://doi.org/10.1007/s00595-009-4242-z" @default.
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