Dmd - treatment

Ataluren is a relatively new treatment for Duchenne muscular dystrophy caused by premature stop codon mutations. We studied the efficacy and safety of ataluren in eleven patients followed at the Queen Silvia Children's Hospital, in Gothenburg, Sweden. This is a retrospective longitudinal study conducted as part of the STRIDE registry. All patients treated with ataluren were followed in a standardized manner, with physical examination, physiotherapeutic evaluation and lung function tests every six months.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research