WEBJanuary 4, 2022. Source: Children's National Hospital. Summary: Medical experts have developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy...
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A Novel Gene Therapy Shows Promise for Limb Girdle Muscular …
WEBMar 3, 2022 · LGMD2B is caused by mutations resulting in the loss of a muscle repair protein, dysferlin. The dysferlin gene is too large to be packaged in an AAV and requires …
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New gene therapy approach offers a potential long-term …
WEBJanuary 5, 2022. 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 …
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Gene therapy offers potential treatment for limb-girdle muscular
WEBJan 4, 2022 · 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 …
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Unmet needs and evolving treatment for limb girdle …
WEBSep 2, 2021 · The disorders discussed include the LGMDs where the most work has been done including calpainopathies (LGMD2A/R1), dysferlinopathies (LGMD2B/R2) and sarcoglycanopathies …
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6-month treatment with galectin-1 improves LGMD2B disease …
WEBMay 23, 2023 · Limb-girdle muscular dystrophy type 2B (LGMD2B) is a devastating autosomal recessive disease for which there are no current FDA-approved treatments. …