Pre U7-53 Study

• Research type

  Research Study

• Full title

  Study of clinical and radiological changes in patients with Duchenne muscular dystrophy theoretically treatable with exon 53 skipping.

• IRAS ID

  122532

• Contact name

  Francesco Muntoni

• Contact email

  f.muntoni@ucl.ac.uk

• Sponsor organisation

  Genethon

• Research summary

  Pre U7-53 is a natural history study. The objective of this study is to monitor the changes in the clinical and radiological outcome in arm muscles of patients with Duchenne Muscular Dystrophy (DMD). This study will be particularly focussing on patients who have a gene misspelling that can be corrected with a specific new technique (i.e. skipping exon 53 of the dystrophin gene).

  The clinical outcomes involve physiotherapy to look at arm muscle strenght and fatigability. Radiological outcomes are looking at the muscle damage caused in the arms of these patients through MRI imaging.

• REC name

  London - Brighton & Sussex Research Ethics Committee

• REC reference

  13/LO/0374

• Date of REC Opinion

  6 Jun 2013

• REC opinion

  Further Information Favourable Opinion