Saturday, March 21, 2015

Potential Treatment for Myotonic Muscular Dystrophy



One of the discoverers of the gene responsible for myotonic muscular dystrophy, Dr. Mani S. Mahadevan, recently discovered a potential treatment for the disease.  The drug that Dr. Mahadevan identified may be able to slow down the progression of muscle damage and muscle dysfunction associated with muscular dystrophy.  The drug is also currently being evaluated for its ability to treat conditions such as rheumatoid arthritis.
            Dr. Mahadevan and his research team used the drug that was identified to conduct laboratory tests on mice with myotonic dystrophy.  After being treated with the drug, the mice showed evidence of improved muscle function and improved grip strength.  Their muscles also became healthier and many of them lived longer.
            The Food and Drug Administration has not approved the drug so it is not yet available for patient use.  Before the Food and Drug Administration approves the drug for patient use, clinical trials must be performed on people with the disease to determine if it is effective in humans.
            I found this study very intriguing.  I think the results of the study show promise for the drug's potential success in the treatment of people with myotonic muscular dystrophy.  I think the findings of the study and the promise for future clinical trials for the use of the drug on humans will provide a lot of hope for people affected by myotonic muscular dystrophy and their families.

3 comments:

  1. This is awesome. Anytime a possible drug treatment for a serious disease comes about, its exciting stuff. A club I'm a part of actually has done some fundraising for the muscular dystrophy association, so ill definitely have to keep up with this.

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  2. Does the article mention anything specific about the drug? I've also done some research on the effect of satellite and stem cells in regenerating muscle tissue (a possible treatment for muscular dystrophy), and I'm wondering if this drug isn't the result of clinical testing of these cells.

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  3. Finding a potential treat for MD and possibly even rheumatoid arthritis is very exciting. Many people are affected by joint problems such as those and if a treatment was found, many people could potentially have decreased or maybe even no pain. Hoping this potential treatment is a success!

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