A breakthrough in Duchenne muscular dystrophy | DuchenneXchange

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A breakthrough in Duchenne muscular dystrophy


Duchenne Muscular Dystrophy (DMD or Duchenne) is a fatal genetic disorder that affects 1 in every 3,500 boys born globally. Duchenne results in progressive muscle weakness due to a mutation in a gene encoding for dystrophin on the X-chromosome. It first affects boys around age four and by age twelve, most people with Duchenne cannot walk. By age 21, most people cannot move below the waist. Few people with Duchenne live past thirty, due to the serious medical complications that include issues with the heart and lung functioning.

Researchers at the Cedars-Sinai Heart Institute offered hope last week to families of kids with Duchenne, when they released the results of the aptly-named “HOPE Duchenne” trial, a joint Stage I/II-A trial that used cellular therapy to treat Duchenne. In the trial, children with Duchenne were given cardiac progenitor cell infusions through a catheter placed in their three main arteries. Cardiac progenitor cells, are a certain type of stem cell that when signaled, develop into new heart tissue.

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