Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up
{{output}}
Duchenne's muscular dystrophy (DMD) is an X-linked neuromuscular disorder caused by deletions (75%), duplications (15-20%) and point mutations (5-10%) in the dystrophin gene. Among the latter, stop-codon point mutations are rare. Female carriers of dystrophin ... ...
