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The LGMD Community Celebrates Adoption of ICD-10 Diagnostic Codes for LGMD

The LGMD Community Celebrates Adoption of ICD-10 Diagnostic Codes for LGMD
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United States , Nicholas Johnson , Paul Melmeyer , Muscular Dystrophy Association , Speak Foundation , Coalition To Cure Calpain , Department Of Neurology , Virginia Commonwealth University School Of Medicine , Jain Foundation , Awareness Foundation , Peter Foundation , Research Fund , Cure Calpain , Vice President , Public Policy , Vice Chair , Virginia Commonwealth University School , Dystrophy Association ,

Gene Therapy for a Rare Muscular Dystrophy Overcomes Earlier Roadblocks

A preclinical study provides proof-of-concept evidence for a new gene therapy approach that offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Researchers at Children’s National have developed the first non-muscle targeted gene therapy that enhances diseased muscle fiber repair and improves muscle function through a single low dose of an AAV vector that produces human acid sphingomyelinase, released by muscle cells immediately following muscle injury. ....

United States , Sreetama Sen Chandra , Jyoti Jaiswal , Daniel Bittel , Jyotik Jaiswal , Eric Hoffmann , Bradley Williams , Children National , Genetic Medicine Research At Children National , Binghamton University , Children National Hospital , Jain Foundation , George Washington University School Of Medicine , Health Sciences , National Hospital , Clinical Investigation , Genetic Medicine Research , National Health System , George Washington University School , Sen Chandra ,