Love, Work, Progress, and Community in Facioscapulohumeral Muscular Dystrophy
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Love, Work, Progress, and Community in Facioscapulohumeral Muscular Dystrophy

Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…

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FSHD: Fulcrum Releases Data from Losmapimod Drug Trial
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FSHD: Fulcrum Releases Data from Losmapimod Drug Trial

by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…

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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy
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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy

A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…

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CRISPR as a Tool to Treat Facioscapulohumeral Muscular Dystrophy
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CRISPR as a Tool to Treat Facioscapulohumeral Muscular Dystrophy

  According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…

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Losmapimod Receives Orphan Drug Designation for the Treatment of FSHD
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Losmapimod Receives Orphan Drug Designation for the Treatment of FSHD

Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…

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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study
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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study

A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…

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Experimental Treatment for Facioscapulohumeral Muscular Dystrophy has Disappointing Results but May Still Benefit Charcot-Marie-Tooth Disease

The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…

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Fulcrum Therapeutics Acquires Experimental Facioscapulohumeral Muscular Dystrophy Drug

According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…

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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation
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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…

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