|Sponsor / Funder||Genethon|
|CI||Professor Francesco Muntoni, Professor Volker Straub|
|Sites||London GOSH, Newcastle|
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