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A Summary of Current and Future Therapies For Gne Myopathy

This is a special report which has been written  by two members from the  gne-myopathy.org website.  They  have summarized various therapies that are currently being tried on patients, and other future therapies that may help patients with gne myopathy. This report is written in a concise and clear form that is easy to understand.  Please  address any query or comments  to the authors whose contact information is at the end of the report. This article could be found on gne-myopathy.org


Therapies for GNE Myopathy: Current Status and Future Prospects 
    A number of approaches for development of a cure for GNE myopathy are currently available. These therapeutic possibilities are being developed for a large number of different diseases, including genetic disorders. None of these have reached a stage where they are ready to be used by patients. Initial studies do indicate that all therapeutic approaches appear to be safe and non toxic, suggesting that these therapeutic platforms can be developed for treatment of different diseases. Sialic acid supplementation as a cure for GNE myopathy is in extended phase two trial and is likely to be the fastest to reach the market. The National Institutes of Health in Bethesda, Maryland, U.S.A. has just started recruiting patients for phase one/phase two trial of Dex-M74, a ManNac substrate.

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