Newly published model of FSHD and a potential gene therapy to improve functional outcomes

Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent dominantly inherited muscular dystrophy in the world. To date, there are no pharmacologic treatments available for the more than 850,000 people affected worldwide. Developing models to use for testing potential therapeutics has been a challenge for the research community.

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