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 DMD 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 Dongsheng Duan, Nathalie Goemans, Shin’ichi Takeda, Eugenio Mercuri and Annemieke Aartsma-Rus discusses all the topics mentioned above.
About this article
Duan, D., Goemans, N., Takeda, S. et al. Duchenne muscular dystrophy. Nat Rev Dis Primers 7, 13 (2021). https://doi.org/10.1038/s41572-021-00248-3
- DOI https://doi.org/10.1038/s41572-021-00248-3
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