I. Richard

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

This study identifies bazedoxifene as a potential treatment for LGMD R2 through high-throughput screening of drugs targeting dysferlin aggregation.

Disease exacerbation by fibroblast inclusion in Duchenne Muscular Dystrophy MYOrganoids reveals limitations of microdystrophin therapeutic efficacy

This paper develops a human DMD model using MYOrganoids to study fibroblast inclusion effects and evaluates microdystrophin therapy efficacy, finding limited success in reducing fibrosis and improving membrane stability.

Context-induced renewal of passive but not active coping behaviours in the shock-probe defensive burying task

This study investigates whether passive and active coping behaviors show renewal after extinction in a shock-probe defensive burying task, finding renewal in passive behaviors but not active ones.

Skeletal muscle cells derived from induced pluripotent stem cells: a platform for limb girdle muscular dystrophies.

This paper describes a platform using human induced pluripotent stem cells to model limb girdle muscular dystrophies and demonstrates its utility in studying disease-specific phenotypes.

Dual blockade of misfolded alpha-sarcoglycan degradation by bortezomib and givinostat combination

This study identifies givinostat as a potential therapeutic for LGMD R3 by inhibiting autophagy and enhancing bortezomib's effect on misfolded alpha-sarcoglycan degradation.

Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in Duchenne muscular dystrophy

This study identifies DD-miRNAs as regulators of mitochondrial dysfunction in DMD, showing their upregulation and epigenetic control in dystrophic muscle.