Muscular dystrophy targeted with promising gene therapy

Child with muscular dystrophy. Image credit: Muscular Dystrophy UK

An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, according to results being presented by the drugmaker Sarepta Therapeutics.

Investors are likely to be focused on a measure of whether a test called the western blot shows that the gene therapy has sufficiently raised levels of dystrophin, the protein that is dysfunctional or missing in Duchenne patients, and without which the body cannot build and maintain muscle.


A May analyst note from Nomura Instinet, an investment bank, predicted the gene therapy would lead to dystrophin levels of 5% to 10% of normal.

The actual western blot number: 38.2% of normal, using Sarepta’s western blot method, and 53.7% based on a method developed at Nationwide Children’s that corrects for readings in fat and scar tissue. In other words, quadruple the best case.

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Sarepta is already planning to start another study of 24 boys. Twelve will receive the gene therapy, and another 12 will receive a placebo. At the end of a year, the 12 patients who received placebo will get the gene therapy, too. The hope is that patients’ function will improve enough that regulators will approve the drug.


Read full, original post: A Gene Therapy Appears To Replace Missing Protein In Muscular Dystrophy Patients

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