Consequently, the aim of the present study was to decipher the pathological mechanism of LGMD2E/R4 in vitro and to validate the repurposing of the pharmacological approach based on CFTR correctors, recently validated in vivo for LGMD2D/R3 [17,21,22,23]. The gene discussed is CFTR; the disease is autosomal recessive limb-girdle muscular dystrophy type 2D.