LGMD2B/Miyoshi Myopathy (collectively called dysferlinopathy) are rare forms of muscular dystrophy caused by mutations in the dysferlin gene. For the disease to occur both copies of the dysferlin gene must be defective (recessive inheritance).
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The Jain Foundation has cultivated a robust dysferlin research field that spans the globe. Read about the active research projects as well as projects from the past and gain an overview of the work being done to discover therapeutics.
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This is a dynamic illustration that will continue to change with increasing knowledge of dysferlin biology and ideas for novel interventions, and how each strategy blocks the progression of disease.
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