Meeting Banner
Abstract #1220


*Valeria Ricotti 1 , *Matthew RB Evans 2 , Christopher DJ Sinclair 2 , Jasper M Morrow 2 , Jordan W Butler 1 , Robert L Janiczek 3 , Michael G Hanna 2 , Paul M Matthews 3 , Tarek A Yousry 2 , Francesco Muntoni 1,2 , and John S Thornton 2

1 Dubowitz Neuromuscular Centre, UCL Institute of Child Health and Great Ormond Street Hospital, London, United Kingdom, 2 MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology, London, United Kingdom, 3 GlaxoSmithKline, London, United Kingdom

Assessing dystrophin restoration in the muscle of DMD patients undergoing experimental gene therapies requires an invasive muscle biopsy. MRI may provide a non-invasive alternative to evaluate response to therapy; however the natural course of DMD muscle changes needs to be established. Examination of the upper limb will allow recruitment of ambulant and non-ambulant patients in clinical trials. Eight non-ambulant DMD patients and 10 healthy controls underwent 3-point Dixon fat-fraction forearm imaging revealing significantly higher fat fraction and muscle atrophy in DMD. Additionally, duration of non-ambulation and wrist extension myometry correlated with MRI indices. MRI is a potential biomarker for evaluating DMD progression.

This abstract and the presentation materials are available to members only; a login is required.

Join Here