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For genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes responsible can help halt the disease.
Earlier today (June 22), the U.S. Food and Drug Administration (FDA) approved the first such intervention, a gene therapy called Elevidys, from Sarepta Therapeutics, a Massachusetts-based biotech company. The approval applies only to children ages 4 and 5, which reflects the conflicting opinions within the agency about the quality of data supporting the gene therapy’s effectiveness, which delayed the decision from the original anticipate date, May 29.
Duchenne is an inherited genetic disorder, more common among males, that affects the muscles, including those not only in the limbs but in the heart and respiratory system as well, causing them to progressively weaken and waste away. The disease is caused by a genetic variant in the dystrophin gene that reduces the amount of dystrophin protein in muscles, making them stiffer and less pliable, eventually causing muscles to contracted too tightly and become less mobile...
