Programme committee

Ségolène Aymé is a medical geneticist, Director of research at the French Institute of Health and Medical Research (INSERM). She is the executive manager of the INSERM department dedicated to information on rare diseases in Paris.

Serge Braun PharmD, PhD has 20 years of experience devoted to neuromuscular diseases in both academic and private laboratories, together with specific expertise in R&D in gene, cell and pharmacology therapies.

Professor Kate Bushby has overseen the expansion of the Newcastle Muscle Centre since 1999 to its position today as a leading international neuromuscular centre.

Dr. Valerie Cwik is Executive Vice President – Research and Medical Director for the Muscular Dystrophy Association (MDA) where she oversees MDA’s extensive research and health care services programs and represents the Association at national and international scientific meetings.

Alessandra Ferlini is a professor in medical genetics, and coordinator of the EU funded BIO-NMD project. Her experience is in the genetics of muscular disorders, and she leads both research and innovative molecular diagnosis projects.

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD) which she founded together with other parents of young men with Duchenne in 1994.

Eric is the Scientific director of CINRG, and has extensive experience with pre-clinical and clinical drug development.

Cynthia Joyce has worked with the SMA Foundation since its inception to build momentum in research and therapeutics development for spinal muscular atrophy. In the first five years of operations, the Foundation facilitated a five-fold increase in research dollars allocated by federal, corporate and non-profit funders and has itself emerged as the leading single sponsor of research in the field.

Dr. Jan Kirschner is  coordinator of the Clinical Trial Coordination Centre (CTCC) based in Freiburg, Germany.

Jerry is the Director of the Centre for Gene Therapy and a Professor of Pediatrics, Neurology, and Pathology with extensive experience in translational neuromuscular disease research including clinical trials.

Francesco Muntoni (Professor in Pediatric Neurology, FRCPCH, FMedSci): Director of the Unit with oversight of both clinical and research activities.

Robert Palm is the President of the Swiss Foundation Progena, which was founded in 2006 shortly after his son was diagnosed with Duchenne.

Program Director for Neuromuscular Disease at the National Institute for Neurological Disorders and Stroke (NINDS), which is an institute of the US National Institutes of Health (NIH).

Professor Volker Straub is joint co-ordinator of TREAT-NMD, executive board member of the World Muscle Society and executive board member of the Institute of Human Genetics at Newcastle University.

Dr Simon Woods is a bioethicist at the Policy, Ethics and Life Sciences Research Centre (PEALS).