Repurposing CFTR corrector to treat muscular dystrophies caused by misfolded α-sarcoglycan
Feb. 7, 2025
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with progressive muscle degeneration. There is currently no approved treatment targeting the underlying cause of the disease.