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GTx-026 Slated as Potential DMD Cure

GTx, Inc recently publicized the results of a study regarding the potential efficacy of their selective androgen receptor modulator or SARM, GTx-026, in terms of treating Duchenne Muscular Dystrophy (DMD).

The study is entitled, “Androgen Receptor Agonists Increase Lean Mass, Improve Cardiopulmonary Functions, and Extend Survival in Preclinical Models of Duchenne Muscular Dystrophy” and was published in the journal Human Molecular Genetics.

The results of the study suggested that the SARM GTx-026 can be a more suitable treatment option for DMD compared to corticosteroids without the side effects arising from the long-term use of the same. Clinical research shows that although corticosteroids are effective in alleviating inflammation, continued use of the same can lead to other health issues like osteoporosis, hyperglycemia and muscle wasting.

How Duchenne Muscular Dystrophy (DMD) affects the muscle cells

Ramesh Narayanan, Ph.D., associate professor, Department of Medicine, University of Tennessee, director of the Center for Cancer Drug Discovery, and a consultant of the biotechnology company based in Memphis, Tennessee, United States, emphasized that GTx-026 is displaying positive effects that not just promise to help extend the survival of boys afflicted with DMD, but also reverse musculoskeletal complications that they may be going through.

Selective androgen receptor modulators or SARMs are basically special ligands that set off beneficial effects akin to the use of anabolic steroids when introduced into the body. These effects include increasing muscle mass and enhancing bone density. These compounds are also seen as potential treatment for stress urinary incontinence in postmenopausal women.

Duchenne Muscular Dystrophy (DMD) affects approximately 18,000 boys between 3 to 5 years old in the United States. It is characterized by musculoskeletal and cardiopulmonary complications and is caused by mutations in dystrophin, which is a protein that helps keep the muscles cells together. It is not uncommon that boys afflicted with this illness are at risk of respiratory and cardiac issues.

If GTx-026’s performance keeps up after the preclinical phase, this SARM could possibly revolutionize DMD treatment.

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