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.
Objectives
- 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