New Phase 1 Study for Becker Muscular Dystrophy Treatment

Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials in San Antonio, Texas.

About Becker Muscular Dystrophy

Becker muscular dystrophy is one of the many forms of muscular dystrophy. This type is characterized by progressive weakness and loss of muscle mass in the cardiac and skeletal muscles. It typically impacts males, with onset occurring between ages five and fifteen. Weakness tends to begin in the pelvis and legs, resulting in symptoms like falling, difficulty walking, and problems with muscle skills. As the heart muscles weaken, cardiomyopathy becomes another symptom. Other effects are fatigue, cognitive problems, scoliosis, difficulty breathing, contractures, a loss of balance and coordination, and muscle weakness in various regions throughout the body. All of these symptoms are the result of a mutation in the DMD gene, which is inherited in an X-linked recessive pattern. There is currently no cure, and treatment is symptomatic.

About the Study

This trial is meant to evaluate the safety, pharmacokinetics, and tolerability of EDG-5506 in both healthy participants and Becker muscular dystrophy patients. This treatment was created in an effort to stop the muscle degeneration that characterizes this disease. It works to stop inflammation and fibrotic response while also promoting physical function and muscle integrity.

It will be double-blind, randomized, single and multiple ascending doses, and placebo-controlled in an effort to investigate the safety, pharmacokinetics, and tolerability of EDG-5506. Participants will be given single or multiple oral doses or a placebo. Find more about this study here.

Researchers are excited about the possibilities that this treatment presents to Becker muscular dystrophy, and possibly Duchenne muscular dystrophy, patients, as they are an underserved population.

Find the source article here.

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