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  • Portrait of Young Girl Patient in Exam Room

    Leader in Care

    Throughout the Washington, D.C, region, the nation and the world, Children’s National Hospital is recognized for our expertise and innovation in pediatric care and as an advocate for all children.

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Latest News

Green test tubes in a lab

New gene therapy approach offers a potential long-term treatment for limb-girdle muscular dystrophy 2B

Children’s National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease. Using a single injection of a low dose gene therapy vector, researchers restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle. The treatment was safe, attenuated fibro-fatty muscle degeneration, and restored myofiber size and muscle strength, according to the study published in the Journal of Clinical Investigation.