Other publications

Workshop reports:

171st ENMC International Workshop: Standards of care and managemnet of facioscapulohumeral muscular dystrophy
Tawil R et al. Neuromusc. Dis. 2008; 18:97-1001

Patient Registries and Trial Readiness in Myotonic Dystrophy - TREAT-NMD/Marigiold International Workshop Report
Thompson R et al. Neruomusc. Dis. 2009; 19(12):860-866

Publications from the national registries:

Dystrophin gene analysis in Hungarian Duchenne/Becker muscular dystrophy families - Detection of carrier status in symptomatic and asymptomatic female relatives
Pikó H et al. Neuromusc. Dis. 2009 Feb. 19:108-112

Characterization of the DMD/BMD patient population in Czech Republic and Slovakia using an innovative approach
Brabec P et al. Neuromusc. Dis. 2009 Apr. 19:250-254

DuchenneConnect Registry Report
Rangel V et al. PLoS Curr. 2012 Feb.

A Modular Approach to Disease Registry Design: Successful Adoption of an Internet-based Rare Disease Registry
Bellgard M.I. et al. Hum. Mut. 2012 Oct. 33(10):E2356-2366

Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?
Scully M.A. et al. Neurol. 2013 Feb. 80(6):583-589

Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients
Takeuchi F et al. J Neurol. 2013 Sept.

Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy)
Nakamura H et al. Orphanet Journal of Rare Diseases. 2013 Apr. 8(1):60


Gene-based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation
Braun S. J. Gene. Med. 2013 Oct

12 Apr 2017