Could Entos Pharmaceuticals’ Fusogenix platform open a real path to treatment in L-CMD?
Entos Pharmaceuticals and the L-CMD Research Foundation are targeting a curative L-CMD therapy. Read what this could change for rare disease treatment.
Can epigenome editing bypass gene size limits? Modalis’ LAMA1 data revives hope in congenital muscular dystrophy
Modalis unveils primate data showing safe LAMA1 gene activation for LAMA2-CMD. Find out what it means for CRISPR therapeutics and CMD patients.