Laurent Servais MD PhD has been a Child Neurologist at the Institute of Myology at Hopital La Pitié Salpêtrière since 2009 and Head of Department of Clinical Trials and Databases at the same institute since 2011. He coordinates the Institute I-Motion since January 2015. Dr Servais graduated from Louvain Medical School in 1999 and received his PhD in Neuroscience from Université Libre de Bruxelles in 2005. He is also the coordinator of the neuromuscular center in Liège, Belgium. His main fields of interest are clinical child neurology, clinical and translational research, and outcome measures development. He has authored or co-authored more than 60 papers in peer-reviewed journals such as New England Journal of Medicine, Lancet Neurology, PNAS, and Molecular Therapy. He was awarded “Young Myologist of the Year’ by the World Muscle Society in 2013.
Charlotte Lilien is a Physiotherapist and moreover a clinical evaluator in many clinical trials at the Institute of Myology at Hopital La Pitié Salpêtrière since 2013. She will be part of the Institute I-Motion team starting in June 2015. Charlotte Lilien has been graduated from the Haute Ecole de la Province de Liège in 2013. Her main fields of interest are clinical child neurology, clinical and translational research, and outcome measures development. She is the principal Physiotherapist for the international Natural History Study in patients with MTM1 which started in May 2014.
Presenting the MTM Natural History Study
A long term study on the natural history of patients with Myotubular Myopathy (MTM1) has been established with the financial Support of Valerion Therapeutics and Genethon.
There is different research running to find therapies for this rare disease and this study has the aim to spport this development.
The goal is to evaluate the history of MTM in a time span between 12 and 36 months. the frequency of the examination depends on age, functional abilities and lies between every three months and once a year.
A extensive range of studies offers Information on strength, Motor skills, respiratory function, liver, kidneys, heart function and quality of life.
The first active open centres are in France (2 in Paris, 1 in Lille, 1 in Lyon und 1 in Toulon). The German centre is in Essen and will open in September 2015.
Other centres will be opened in London and Rome . The Study will also take place in the USA and Canada. A total of 60 patients should be involved, 40 in Europe and 20 in North America.