Study: Gene Therapy as a Treatment for Duchenne Muscular Dystrophy

According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional measurements. As of now, researchers believe that this therapy is better than the current standard of care.

About Duchenne Muscular Dystrophy

DMD is one of nine forms of muscular dystrophy and is characterized by progressive muscle weakness. It occurs when the body is unable to make dystrophin, which is due to an X-linked recessive mutation. Symptoms will manifest within the first six years of life, and they include frequent falling, fatigue, difficulty with motor skills, learning disabilities, issues with walking and moving positions, muscle weakness that starts in the legs and pelvis that progresses throughout the body, and eventually heart disease and respiratory failure. Doctors will use a variety of tests to diagnose this condition, including EMGs, muscle biopsies, CPKs, and genetic testing. After a diagnosis is obtained, treatment is aimed at slowing progression and managing symptoms. This includes steroids, heart medicine, assisted breathing, amino acid treatment, asthma medication, supplements like creatine, and physical and occupational therapy.

About SRP-9001

SRP-9001 is a gene therapy that sees the systemic delivery of micro-dystrophin to the nuceli of cells. It was studied in a clinical trial in patients ranging from four to seven years old. They were treated with one dose of SRP-9001, administered via infusion. While there were treatment-related events, they were mild to moderate in severity; the most common event was vomiting.

12 weeks after being dosed, researchers performed muscle biopsies and found that 81.2% of muscle fibers were expression micro-dystrophin. This positive result was joined by improvements on the North Star Ambulatory Assessment (NSAA). Participants also saw a reduction in levels of creatine kinase and improvements in other functional tests, such as climbing stairs and sitting and standing up.

This study is still ongoing, and it is expected to end in March of 2021. Researchers hope that the positive results continue, as SRP-9001 would improve the current standard of care and the lives of those with DMD.


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