TREAT-NMD Executive Committee
The TREAT-NMD Executive Committee, made up of 9 academic representatives and 4 patient representatives, took over governance of the network in 2012. The members of the Executive Committee were elected from the TREAT-NMD task force and from member patient organisations. The members of the committee then elected their Chair and Vice-Chair by majority vote.
The TREAT-NMD task force, intially set up to oversee the transition from the network's EU funding status, was asked to nominate and vote for the scientific contingent of the committee. Over 120 patient organisations from 51 different countries were involved in the voting process to determine the three patient organisation representatives that complete the committee.
Vice-Chair Eric Hoffman was scheduled to take over as Chair of the Executive Committee of the TREAT-NMD Alliance from Annemieke Aartsma-Rus in December 2015 during the TREAT-NMD International Conference. However, Eric has recently taken a year leave of absence from his academic position to aid drug development and clinical trial efforts in the private sector. To avoid perceived or real conflict of interest, Eric voluntarily stepped down as Vice Chair of the TREAT-NMD Executive Committee, and thus will not assume the Chair position in December. Eric will remain a member of the TREAT-NMD Executive Committee.
The TREAT-NMD Charter did not specify whether individuals employed by industry can be Executive Committee members. The Executive Committee has discussed this matter and has voted on it. The outcome is that it is acceptable to have individuals employed by industry as members of the Executive Committee; however, they are precluded from taking (Vice)-Chair positions while being employed by industry.
In light of these developments, a new Vice-Chair was required. We are pleased to announce that Kevin Flanigan has stepped up as the new Vice-Chair. He will take over as Chair from Annemieke in October 2016, when Filippo Buccella will become the new Vice-Chair.
The TREAT-NMD Alliance is governed by the charter which can be found here.
Professor of Pediatrics and Neurology - Ohio State University College of Medicine
Dr. Kevin Flanigan is a Professor of Neurology and Pediatrics at the Ohio State University, and a Principal Investigator at the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio. He has extensive experience in the design and development of patient registries, and has served as the Director of the United Dystrophinopathy Project, an NIH-funded genotype/phenotype database and registry.
Italian DMD Registry Curator - Parent Project - Genitori contro la Distrofia Musculare di Duchenne & Becker ONLUS Italy
As father of a young man affected by DMD, Filippo has been working since 1996 to advocate for Duchenne and Becker patients in Italy, Europe and worldwide, always convinced that their work could open a new path for many other similar diseases/conditions. Filippo is also a pharmacist and his knowledge of the mechanisms of action of drugs and/or drug development are very helpful when it comes to translating complicated topics for families and patients. He has been involved with patient registries from the very beginning and have set up a national DMD/BMD patient registry in Italy, which is has been instrumental for conducting clinical trials.
In his role as president of DPP-Onlus Italy he has been able to establish a good working relationship with many other organizations worldwide over the years. This network provides an invaluable asset for a member of the Executive Committee of the TREAT-NMD Alliance.
Filippo is keen to continue to bring the voice of patients as key stakeholders to the TREAT-NMD Alliance. He also aims to collaborate with patient organizations to elicit their suggestions/expectations and to enable him to represent the wider patients’ view in the committee.
Professor - Leiden University Medical Center
Dr. Annemieke Aartsma-Rus played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy during her PhD research (2000-2004), which was supervised by Dr. Judith van Deutekom, Dr. Johan den Dunnen and Prof. Dr. Gert-Jan van Ommen at the Leiden University Medical Center, Department of Human Genetics (the Netherlands). She successfully defended her thesis titled “Development of an antisense-mediated exon skipping therapy for Duchenne Muscular Dystrophy – Making sense out of nonsense” on February 10th 2005. Since September 2004 she continued this research as a post doc under supervision of Dr. Judith van Deutekom.
M.D - UZ Leuven
Nathalie Goemans, MD, PhD, is a paediatrician and child neurologist, with certification in rehabilitation medicine. She is head of clinic within the department of Pediatric Neurology and head of the Neuromuscular Reference Centre for Children at the University Hospitals Leuven. She has an academic position at the University of Leuven, Belgium. She is vice chair of the TGDOC within TREAT- NMD.
Nathalie has a particular interest in the multidisciplinary aspects and the dissemination of standards of care in neuromuscular disorders. Research activities involve therapy development and development of outcome measures to assess interventions in these diseases.
Director, Research Center for Genetic Medicine & The Wicker Project for Muscular Dystrophy Research - CINRG
Eric Hoffman, PhD earned his PhD in Genetics at Johns Hopkins University and then pursued post-doctoral training with Louis Kunkel at Boston Children’s Hospital working on Duchenne muscular dystrophy gene and protein identification.
Consultant Pediatric Neurologist - University Medical Center Freiburg
Dr. Jan Kirschner is coordinator of the TREAT-NMD Clinical Trial Coordination Centre (CTCC) based in Freiburg, Germany. Jan has been joint coordinator of the German network for neuromuscular disorders MD-NET (www.md-net.org) since 2008. He has been involved in planning and conducting several multicentre clinical trials. He was responsible for the coordination of a large multi-centre, placebo-controlled MD-NET trial in Duchenne muscular dystrophy. With the CTCC team in Freiburg Jan has established the Care and Trial Site Registry (CTSR) containing feasibility information about neuromuscular centres worldwide.
Professor of Pediatric Neurology - Università Cattolica del Sacro Cuore
Eugenio Mercuri Md PhD (Associate Professor in Pediatric Neurology): areas of expertise: congenital muscular dystrophy, outcome measures and assessment tools in neuromuscular disorders.
Director, Department of Neuromuscular Research - National Institute of Neuroscience - National Center of Neurology and Psychiatry
After having 5 years of clinical neurology training, Dr. Nishino started muscle disease research from 1994 under the tutelage of Dr. Ikuya Nonaka, who remained a steadfast source of inspiration. Later, his postdoctoral research for two years at Columbia University solidified his yearning to focus on research. In 2001, he was appointed directorship of the current Department of Neuromuscular Research and since then, he has been directing his team on the research to elucidate the pathomechanism and develop the therapy for hereditary muscle diseases, including DMRV/hIBM and various muscular dystrophies.
President of Nonprofit-organization PADM - Patient association for distal myopathies
Yuriko was diagnosed with GNE Myopathy in 2002 and founded PADM, the Patients Association for Distal Myopathy in 2008. Japanese scientist Dr. Ichizo Nishino proved the effective material for GNE Myopathy, however there were no pharmaceutical companies interested in further development of a medicine. PADM repeatedly petitioned against this finally reversing this decision in Japan in 2010 followed by the US and other countries.
Since April 2008, PADM has been campaigning for distal myopathy to be recognised as a designated rare disease in Japan. After collecting over 2 million signatures they achieved their goal in 2015.
PADM wishes to overcome difficulties and disabilities not only for people with distal myopathies, but for all kinds of patients. They have recently started to build a new system for barrier-free map in 2015, when they received the grand prize in Google Impact Challenge in Japan.
Chairman of the Swedish Research Foundation for Muscular Dystrophy and Member of the board of the Association for Disable Children and Adolescents
Since the beginning of 2014 Petra has been the chairman of the Swedish non-profit patient organisation Swedish Research Foundation for Muscular Dystrophy, SMDF as well as a board member and responsible for the MD section of the Association for Disable Children and Adolescents.
Petra holds a holistic perspective on the situation for a family with a Duchenne diagnosed child/adolescents. This includes caregivers, school, municipalities, family and relatives. This engagement has resulted in Swedish media coverage and Petra continues to work for an overall better situation especially for the DMD community but also disable children/adolescents in general. Petra has also been involved in the process for having the DMD treatment Translarna/Ataluren getting out to the targeted patients in Sweden.
Privately Petra runs her own company working professionally with being the chairman on company boards of directors for several Swedish high growth high tech companies. She lectures at Gothenburg University, Chalmers University of Technology and is accredited teacher at the Swedish Academy of Board Directors.
Petra is the mother of Max who is born 2007 and was diagnosed with DMD year 2011 and Attention Deficit Hyperactivity Disorder (ADHD) year 2014. The reason for Petra to be a part of a larger MD community is to make a difference for this community internationally.
The Rehabilitation Centre for Neuromuscular Diseases
Jes is a specialist in neuromuscular rehabilitation and also a volunteer in Muskelsvindfonden, the Danish patient organization for NMD. Additionally Jes is Chairman of Muskelsvindfonden's 1) scientific committee, 2) ethical committee and 3) international relations committee.
He represents Muskelsvindfonden in the ENMC where he has just been elected Chairman of the Executive Committee.
Jes works as Chief Medical Officer and Director of the Danish National Rehabilitation Centre for Neuromuscular Diseases and is an associated partner in CARE-NMD which is a DMD project under TREAT-NMD.
Associate Professor, Astrid Lindgrens Barnsjukhus - Karolinska Institute
Thomas Sejersen holds positions as Professor in Neuropediatrics at the Department of Women's and Children's Health of Karolinska Institute and consultant paediatrician at the Astrid Lindgrens Barnsjukhus (Stockholm, Sweden).
Consultant, Hendaye / Head, Summer School of Myology, Paris - Hôpital Marin de Hendaye
Dr Jon Andoni Urtizberea is head of the internationally renowned Paris Summer School of Myology and a consultant at the neuromuscular reference centre at Hendaye Hospital in the French Basque region.