New Drug Targets Identified for Potential Use in Development of Treatments for Muscular Dystrophy

Posted on January 12, 2017 by Disability Help Group

Researchers at the University of Basel have identified two pathways that hold a potential for use in developing new drug targets for muscular dystrophy treatment medications. The study looked at the AMPK and mTOR signaling pathways that regulate the energy balance and autophagy in cells.

When a person has myotonic dystrophy type I (MD1), a common form of muscular dystrophy, poor gene signaling disrupts these pathways, which causes many of the symptoms of the disease. After researchers used treatments designed to clear the pathways, MD1 symptoms improved in mouse models.

When researchers introduced a drug that activates AMPK signaling, it improved muscle function and reduced the abnormal gene splicing that causes the accumulation of toxic gene transcripts. These transcripts build up in muscle cells and cause muscle wasting and weakness. Rapamycin, an existing drug approved for activating mTORC1 signaling, reduced signs of muscle wasting.

Researchers published the study in JCI.

Disability Benefits Cover Many Genetic Diseases

Muscular dystrophy is one of several genetic diseases that have no known cure and can prevent individuals from working and earning a substantial income. Because muscular dystrophy is a progressive disease, most people who develop it become unable to work before they hit retirement age.

If you have worked for part of your life and now can no longer continue working due to your health conditions, call the Disability Help Group at 800-800-2009 to discuss your case with a disability advocate.