Updates On Duchenne Muscular Dystrophy Treatment

Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001, a treatment of Duchenne muscular dystrophy.

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy is one of the nine forms of muscular dystrophy. Affected individuals experience progressive muscle weakness and wasting due to a lack of dystrophin. Symptoms include issues with motor skills, muscle weakness, falling, fatigue, issues with changing positions, problems walking, learning disabilities, and eventually heart disease and respiratory failure. All of these effects are caused by a mutation passed down on the X chromosome in a recessive pattern. There is currently no cure for this condition, but treatments can help to manage symptoms.

Updates on SRP-9001

Sarepta had to meet with the FDA in order to receive their concurrence on the beginning of their next clinical trial of SRP-9001, a gene transfer therapy, as they want to use commercial process material.

In this meeting, the FDA requested that there be another potency assay for release before participants are dosed. While Sarepta already has data on potency assays, more dialogue needs to happen to figure out if this data can be used.

There were other requests from the FDA, and Sarepta is excited to work with them in order to provide the best treatment that they can to those with Duchenne muscular dystrophy.

Read the source article here.

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