Pharmacological advances for treatment in Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of dystrophin, essential for muscle fibre integrity. Despite extensive pre-clinical studies, development of an effective treatment has proved challenging. More recently, significant progress has been made w...

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Hlavní autoři: Guiraud, S, Davies, K
Médium: Journal article
Jazyk:English
Vydáno: Elsevier 2017