ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial

According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy WVE-210201 will warrant continued testing of the drug in future trials. WVE-210201 is in development as a treatment for Duchenne muscular dystrophy in patients who qualify for axon-skipping. Wave Life Sciences is dedicated to the development of innovative treatments for genetic diseases.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.

WVE-210201 and Exon Skipping

The company plans to initiate further trials next year. WVE-210201 is also continuing to be studied in a ongoing open label extension study of patients that have completed the Phase 1 trial. The genetic origins of Duchenne muscular dystrophy are not the same for each patient. In about ten percent of patients, an approach called exon skipping can be a useful therapeutic target, and it allows for dysfunctional portions of the dystrophin gene to be “skipped” as it gets transcribed to RNA. Therapies the utilize exon skipping allow patients to produce a more functional version of dystrophin, which reduces their symptoms.

While this approach will not work for all patients with Duchenne muscular dystrophy, the advancement of exon skipping approaches nevertheless offers the potential for major improvements in outcomes for patients whose disease is susceptible to it.

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