Clinical and radiological changes in teens with DMD theoretically treatable with exon 53 skipping

Sponsor / Funder	Genethon
CI	Professor Francesco Muntoni, Professor Volker Straub
Sites	London GOSH, Newcastle
Contact details	ich.neuromusculartrials@ucl.ac.uk

This is a natural history study. This study will recruit DMD patients who are treatable with AAV-mediated exon 53 skipping.

to monitor the clinical and radiological course of arm muscles impairment in those patients
to assess serum and urine biomarkers to monitor non-invasively disease progression
and to assess prevalence of immunity against adenoviral vectors in these patients