Gene Therapy Could Be a New Treatment for Muscular Dystrophy

The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was developed by Nationwide Children’s Hospitals.

Muscular dystrophy is a group of diseases that result in the breakdown of skeletal muscles over time. There are multiple sub-types that vary in how rapidly symptoms occur and which muscles are affected. In most cases, other body systems are usually affected, particularly if the muscles necessary for them to function are degenerated. If the dystrophy affects breathing muscles or the heart, it can shorten lifespan.

While still in its infancy, gene therapy offers potential treatments for a variety of illnesses, with muscular dystrophy being just one of them. Source: pixabay.comThe financing should allow Myonexus to proceed with the next stage of its clinical trials, which is specifically targeted towards the treatment of limb-girdle muscular dystrophy. There are over thirty types of limb-girdle muscular dystrophy; this kind often affects muscles of the hips and shoulders, and most people with the illness will lose their walking ability within twenty years of diagnosis.

Since muscular dystrophy is genetically inherited, the gene therapy approach seems like a good fit for treatment. In gene therapy, nucleic acid is delivered into a patient’s cells as a drug to treat disease. Interestingly, this is done with a genetically engineered virus, which replaces the gene that causes the illness with one that does not.

This trial is intended to test the third genetic variation that Myonexus has licensed. While it hopeful that the trials will demonstrate the effectiveness of gene therapy treatment, it will still take some time before the new treatments will be close to regulatory approval and usage by the public. The need for treatment is urgent however, as currently there are no treatments that are approved for limb-girdle muscular dystrophy.

The lack of treatment options available for people suffering from limb-girdle muscular dystrophy means that many will be looking closely at the results of the clinical trials.

Much of current medical care for muscular dystrophy involves only attempts to manage the symptoms of the disease, including efforts to slow down muscular degeneration or delay damage to dying muscle cells. The advent of gene therapy will represent a shift in merely addressing symptoms to attempting to resolve the causes of the disease.


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