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Open Access: Recent studies on Duchenne muscular dystrophy

March 15, 2021
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Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for Duchenne that address obtaining a genetic diagnosis and managing the various aspects of the disease have been established.

In addition, a number of therapies that aim to restore the missing dystrophin protein or address secondary pathology have received regulatory approval and many others are in clinical development.

This open-access Nature article by Defeat Duchenne Canada funded researcher, Dongsheng Duan, along with Nathalie Goemans, Shin’ichi Takeda, Eugenio Mercuri and Annemieke Aartsma-Rus discusses all the topics mentioned above.

Click here to read the full article on Nature.com

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