FDA Approves Gene Therapy for Muscular Dystrophy in Children

Treatment is intended for kids with a confirmed mutation of the DMD gene.

The U.S. Food and Drug Administration (FDA) recently approved Elevidys, the first gene therapy for treating children between the ages of four and five with Duchenne muscular dystrophy (DMD). The treatment is for child patients who have a confirmed mutation in the DMD gene and no medical reasons preventing them from receiving the therapy. DMD is a rare and progressive genetic condition that causes muscle weakness and deterioration over time.

“Today’s approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual’s health over time,” said FDA’s Center for Biologics Evaluation and Research Director Peter Marks. “The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”

As the Lord Leads, Pray with Us…

  • For Commissioner Califf to seek God’s wisdom as he heads the Food and Drug Administration.
  • For FDA researchers as they review and test treatments and therapies for authorization.
  • For U.S. health professionals as they treat patients with chronic illnesses and diseases.

Sources: Food and Drug Administration


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