Article

Identification of bazedoxifene for the treatment of LGMD R2 by high throughput screening

2024-03-04

Abstract excerpt

LGMD R2 is a rare genetic disorder characterized by progressive proximal muscle weakness and wasting caused by a recessive loss of function of dysferlin, a transmembrane protein controlling plasma membrane repair in skeletal muscles. We report here the development of an in vitro high-throughput assay using immortalized myoblasts and monitored reallocation of an aggregated mutant form of dysferlin ( DYSF L1341P )...

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Literature Corpus work: ba39649a-e391-588f-9a47-475358ebaabc
DOI: 10.1101/2024.02.28.582550

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