"[TACT] comments highlighted relevant issues of the proposal and will surely help us to strengthen the final version of the project."
Professor Giuseppe Vita &
Dr Sonia Messina
University of Messina, Messina, Italy
Kathryn Wagner, as Chair, is leading this exciting initiative with the support of the core committee, nominated extended committee members and the TREAT-NMD secretariat.
In order to oversee the work of TACT on a regular basis and to provide guidance on its structure and content, a "core" group serves as a driver and common denominator of a broader membership.
Working closely with the Chair and core group are >70 additional international multidisciplinary experts, each with specific expertise in one or more of the areas of drug development. Such expertise include: discovery research, preclinical assessment of animal and cellular models, chemistry, preclinical pharmacology, formulation, toxicology, biostatistics, regulatory expertise in both the US and the EU, clinical care, clinical research, clinical development, clinical trial expertise, ethics, patient advocacy and funding of development programmes.
TACT is supported by the TREAT-NMD secretariat and importantly, patient organisation representation who help with the selection of projects, as part of the review panel, and in providing part funding for meeting costs. All members of TACT are required to sign a common confidentiality and disclosure agreement, binding for all of the information received from applicants, and conflict of interest statements are updated on a meeting-by-meeting basis.
TACT is supported by the TREAT-NMD secretariat and importantly, patient organisation representation who help with the selection of projects, as part of the review panel, and in providing part funding for meeting costs.
All members of TACT are required to sign a common confidentiality and disclosure agreement, binding for all of the information received from applicants, and conflict of interest statements are updated on a meeting-by-meeting basis.
Associate Professor of Neurology - The Johns Hopkins School of Medicine
Kathryn R. Wagner, M.D., Ph.D. is the Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and Associate Professor of Neurology and Neuroscience at the Johns Hopkins School of Medicine. She treats patients with muscular dystrophies in a multidisciplinary clinic, addressing the multiple medical and social issues affecting these individuals and families.
Head of Regulatory Affairs and Quality Assurance - Généthon
Didier is a pharmacist specialised in Regulatory Affairs and also in product development. Since 2001 he has worked for GENETHON where he has successfully obtained Orphan Drugs designations and Clinical Trial Authorisations for gene therapy products in the field of NMD in several European countries.
Principal - Csimma LLC
Cristina Csimma’s experience in the biopharmaceutical, venture capital and academic settings encompasses global development in multiple therapeutic areas including rare neuromuscular diseases, translational through registration clinical studies, and integration of biomarkers and novel technologies into development as well as leadership roles in new company formation and fund raising.
Professor - University of Western Australia
Miranda has devoted over 30 years of research to better understanding the process of damage and repair of normal skeletal muscle.
Chief Scientific Officer - CureDuchenne
Dr. Michael Kelly is Chief Scientific Officer of CureDuchenne. Dr. Kelly, a senior pharmaceutical executive, brings more than 25 years of experience in drug discovery and development to the organization. He is responsible for advancing drug development programs and identifying new drug targets that exhibit potential to transform the treatment of Duchenne muscular dystrophy.
President, PharMac LLC - PharMac LLC
John McCall’s expertise is drug discovery, risk minimization, and medicinal chemistry. He has worked in a number of disease areas including acute and chronic neurologic disorders, psychiatric diseases, neuromuscular disease, cardiovascular disease, inflammatory disease, and oncology. He is currently President, PharMac LLC.
Director, Division of Clinical Innovation - NCATS
Petra Kaufmann, M.D., M.Sc., is Director of the Office of Clinical Research (OCR). In this capacity, she oversees the clinical research programs funded by the Institute. The OCR fosters clinical research that increases our understanding of the cause, diagnosis, treatment, and prevention of neurological diseases and translates scientific discoveries into improved therapies for people living with neurological diseases worldwide.
Professor of Pediatrics & Child Neurology - Universite Catholique de Louvain
Gunnar Buyse (MD, PhD) is Professor of Pediatrics and Child Neurology at the University of Leuven (Katholieke Universiteit Leuven), and is deputy clinical chair of Child Neurology at the University Hospitals Leuven. Gunnar Buyse is a Committee Member of the TREAT-NMD Advisory Committee for Therapeutics (TACT).
Professor of Pharmacology - University of Bari
Annamaria is a pharmacologist specialized in pre-clinical in vivo and ex vivo studies of inherited and acquired neuromuscular disorders. In this general frame, she has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field.
Professor - Leiden University Medical Center
Dr. Annemieke Aartsma-Rus is associate professor at the Department of Human Genetics and leader of the DMD genetic therapy group. She is currently Chair of the executive committee of the TREAT-NMD alliance and is a member of the Project Ethics Council and TACT Committee.
Professor of pediatrics and medicine - Texas Childrens Hospital
Hugh's background is in echocardiography and cardiac function analysis, coupled with a large experience with the muscular dystrophy population. Presently he is a Professor of Paediatrics at Baylor College of Medicine, and is Staff Cardiologist at The Heart Center at Texas Children’s Hospital where he directs the care of neuromuscular cardiology patients.
Consultant Cardiologist - Freeman Hospital
John graduated from the University College Dublin, Ireland in 1979. He is currently a Consultant Cardiologist (Electrophysiologist) at the Department of Cardiology, Freeman Hospital, Newcastle upon Tyne, UK, Senior Lecture at Newcastle University & an Associate Member of Institute of Human Genetics, International Centre for Life, Newcastle.
Professor - Ohio State University College of Medicine
Arthur completed his BS in Cellular & Microbial Biology in 1978 at the University of Calgary and his PhD in Biochemistry at University of London.
Head of the School of Pharmacy and Life Sciences (PALS) - Robert Gordon University
Professor Donald Cairns obtained his Pharmacy degree and PhD from the University of Strathclyde. Following a year as a post-doctoral research fellow with the Cancer Research Campaign, Donald moved to Leicester Polytechnic where he held a five-year lectureship in pharmacy. In 1992 Donald was appointed senior lecturer in medicinal chemistry in Sunderland School of Pharmacy and in 2003 moved to Robert Gordon University in Aberdeen as Associate Head. In 2006, he was promoted to Professor of Pharmaceutical and Medicinal Chemistry, in 2010 he was appointed Acting Head of School and in 2012 he was appointed to his present post of Head of the School of Pharmacy and Life Sciences.
Professor of Neurology - University of Pittsburgh
Paula has a career-long interest in the muscular dystrophies spanning clinical care, basic research and clinical research endeavors. She is experienced as a site PI and study chair for studies conducted by the multi-center clinical trial network comprised of more than 25 centers worldwide, the Cooperative International Neuromuscular Research Group (CINRG).
Chief, Division of Biostatistics and Study Methodology - Childrens National Health System
Avital earned her PhD in Statistics at Carnegie-Mellon University. She has specialized in clinical research and particularly in multi-center clinical trials. She is the Director of the Coordinating Center of the Cooperative International Neuromuscular Research Group (CINRG), a network of more than 20 clinical sites focusing on improving the quality of life of patients with neuromuscular diseases and their families via research. She has also directed clinical trials coordinating centers in epilepsy, premature infants and other diseases.
Independent consultant - based in the UK
Simon is a medical statistician with around 30 years’ experience working in clinical trials. He has worked in many different therapeutic areas and has been involved in academic-run studies as well as many carried out by the pharmaceutical industry. Apart from trial expertise, he also has a lot of experience in the overall drug-development process.
Board of Directors - Association Française contre les Myopathies - AFM
Beatrice de Montleau is the mother of three children, the youngest of whom is affected by Duchenne Muscular Dystrophy. Beatrice began her volunteer involvement with the AFM (French Muscular Dystrophy Association) by serving on the financial committee, bringing to that service a professional background in internal audit and sustainable development.
Neurologist, Professor of Neuromuscular Disorders - Amsterdam Medical Centre
Marianne has been the leader of the neuromuscular group at the Academic Medical Centre in Amsterdam in the Netherlands for more than 20 years. During this time her group has contributed to research on muscular dystrophies, myositis, motor neuron diseases and hereditary neuropathies.
Head of the Department of Pediatric Cardiology - Children's Hospital, University Hospital Erlangen
Prof. Sven Dittrich, MD is principal investigator of the German multicentric trial “Effect and Safety of preventive Treatment with ACE-Inhibitor and Beat-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy”, which is organized within the MD-Net and the German Competence Network for Congenital Heart Disease. His group also works on Tissue Doppler and cardiac MRI in Duchenne patients.
Director of Dept of Neurology and Director Neuromuscular Unit - Fundacion Favaloro Hospital Universitario
Alberto Dubrovsky, MD, is currently Director of the Department of Neurology, Director of Neuromuscular Disease Unit of the Neurosciences at Fundacion Favaloro. Since 1983 he has also served as Medical Director of the Argentina Muscular Dystrophy Association. Alberto is a Professor of Neurology at the University of Buenos and a Full Professor of Neurosciences at the Favaloro University, training as a Neurologist in the field of Neuromuscular Diseases at NIH under the sponsorship of W King Engel.
Consultant Physiotherapist - Newcastle University
Michelle initially specialised in Rheumatology and gained a masters degree in the clinical evaluation and development of outcome measures in Ankylosing Spondylitis.
Branch Chief - National Institute of Neurological Disorders and Stroke
Kenneth is Chief of the Neurogenetics Branch at NINDS and a TREAT-NMD STAC member. He has experience with preclinical (animal) studies in various neuromuscular diseases including Duchenne muscular dystrophy, spinal muscular atrophy, and spinal and bulbar muscular atrophy (SBMA), and he has run clinical trials and biomarker studies in Duchenne muscular dystrophy, spinal muscular atrophy, Friedreich's ataxia, and SBMA.
Professor of Pediatrics and Neurology - Ohio State University College of Medicine
Kevin is the Director of the United Dystrophinopathy Project, an NIH-funded consortium studying the natural history and genotype/phenotype correlations in the DMD gene.
CEO - Parent Project Muscular Dystrophy
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.
M.D - UZ Leuven
Dr Nathalie Goemans (M.D. PhD) is a paediatrician and child neurologist, with certification in rehabilitation medicine and is currently head of the Neuromuscular Reference Centre within the department of Paediatrics and Child Neurology at the University Hospitals Leuven, Belgium, and consultant in neuropediatrics at DVC St Jozef Antwerp, Belgium, a rehabilitation centre with residential setting for neuromuscular patients.
Program Director at the National Institute of Neurological Disorders and Stroke
Amelie Gubitz serves as a Program Director at the National Institute of Neurological Disorders and Stroke (NINDS), USA. Her portfolio includes basic, translational and clinical research on amyotrophic lateral sclerosis, X-linked spinal and bulbar muscular atrophy and hereditary spastic paraplegia. Amelie earned a Ph.D. in pharmacology from the University of Cambridge, UK, and then completed a Wellcome Trust Fellowship at Harvard Medical School in circadian biology. Subsequently, she worked as a Senior Scientist in the target discovery group for neuropathic pain at Parke Davis, UK. Prior to joining the NINDS in 2006, she studied the molecular pathogenesis of spinal muscular atrophy in the laboratory of Dr. Gideon Dreyfuss at the University of Pennsylvania.
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 - Lakeside Regulatory Consulting Services Ltd
Joseph Irwin became a Member of Spinal Muscular Atrophy Support UK (previously The Jennifer Trust for Spinal Muscular Atrophy) after his first son Toby was diagnosed with Type I SMA. Joseph, who is a biochemist-pharmacologist with molecular biology and cell biology experience, has worked for many years in hospital based research and for the pharmaceutical industry.
Research Director - Cure SMA
Jill has worked as the Families of SMA (FSMA) research director since 2005. In this capacity, she has overseen over $35 million in FSMA research investments, including in basic research, preclinical drug development, and clinical research.
Director, Neuropediatrics and Muscular Disorders - University Medical Center Freiburg
Prof. Rudolf Korinthenberg, MD is head of the Department of Neuropediatrics and Muscular Disorders at Children’s Hospital, University Hospital Freiburg. He received his pediatric and neuropediatric training at University Hospital Münster/Westfalia and holds his actual position since 1990.
Assistant Professor - University of Massachusetts Medical School
Dr Oliver King is an assistant professor in the Cell & Developmental Biology (primary), and Neurology departments of University of Massachusetts Medical School.
Research and Development / Drug Discovery
Dr Donald Kirsch has almost 35 years of R&D leadership and management experience in the biopharmaceutical industry. He currently runs a biopharma consulting practice in Boston and New York, teaches Drug Discovery and Management at the Harvard University Extension School and courses at MassBio.
Director of Clinical Therapies Research - Nationwide Children's Hospital
Linda Lowes, PT, PhD is the director of clinical therapies research and a member of the Center for Gene Therapy at the Research Institute of Nationwide Children’s Hospital.
Associate Professor, Orthopaedics - University of Maryland - School of Medicine
Rich has been a licensed physical therapist for over 20 years. He received his PhD in 2003, which was aimed at elucidating the cellular and molecular changes that occur during eccentric injury to muscle. He performed his post-doctoral work in the Department of Physiology at the University of Maryland (UMB) School of Medicine where he examined the role of intermediate filaments in skeletal muscle. Rich now runs a lab in the Department of Orthopaedics at UMB. He still maintains a strong interest in measuring muscle contractility and his most recent research tries to relate the changes identified in the lab at the cell and whole muscle levels to changes seen using non-invasive MR imaging and spectroscopy.
Medical Director, Pulmonary Function Testing Laboratory - Children's Hospital of Philadelphia
Hank is a pediatric pulmonologist with a strong interest in the pulmonary manifestations of neuromuscular disease. He has a special interest in chest wall and spinal disease and has helped develop the Center for Thoracic Insufficiency Syndrome at The Children's Hospital of Philadelphia.
Consultant Research Physiotherapist - Newcastle University
Dr Anna Mayhew is a Consultant Research Physiotherapist at The MRC Centre for Neuromuscular Diseases at Newcastle, Newcastle University.
Chair and Director - University of Chicago
Elizabeth McNally is Chair and Director at Center for Genetic Medicine, Northwestern University Feinberg School of Medicine.
National Institutes of Health - NIH
Dr. Elizabeth McNeil came to NINDS in 2010, after working for 8 years at the US Food and Drug Administration (FDA). While at the FDA, she worked extensively on a full range of products, including new molecular entities, new formulations of approved drug products, drug-drug combinations, and drug-device combinations. She worked to develop new scientific, clinical and regulatory standards for the development of certain drug classes and developed initiatives which resulted in standardization and clarification of terminology used in defining claims and endpoints, as well as in increased standardization, precision, and sensitivity in reporting safety and efficacy data.
Founder and CEO - CureDuchenne
Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne. She relies on her extensive background in sales and marketing to lead CureDuchenne.
Director of The Dubowitz Neuromuscular Centre - University College London
Francesco Muntoni (Professor in Pediatric Neurology, FRCPCH, FMedSci) is Director of the Unit with oversight of both clinical and research activities.
Professor of Integrative Systems Biology and Pediatrics - Childrens National Health System
Kanneboyina Nagaraju, DVM, PhD is a principal investigator at the Research Center for Genetic Medicine, Children’s National Medical Center (CNMC) and a tenured associate professor of Integrative Systems Biology and Pediatrics at George Washington University School of Medicine and Health Sciences.
Board of directors - Association Française contre les Myopathies - AFM
Marie-Christine is the mother of a girl affected by SMA. She is a member of AFM Board of Directors, AFM international committee and TREAT-NMD Project Ethics Council. She is also Vice President of SMA Europe (association of European SMA patients)
Pharmaceutical Research and Development Executive - Mike Pleiss and Associates, LLC
Mike was part of several project teams (and head of the multiple sclerosis (MS) / autoimmune effort) that resulted in several first-in-class therapeutics for Alzheimer’s (AD), MS and other autoimmune diseases.
Director of Research - Muscular Dystrophy UK
Marita is the Director of Research at Muscular Dystrophy UK which has funded high quality research into neuromuscular disorders for the last 50 years. She in charge of an annual £1.2m budget invested to increase understanding of muscle function and muscle disease and to ensure an efficient bench-to-bedside transition of promising technology.
Chief Executive Officer - Parent Project Muscular Dystrophy
John Porter is Chief Executive Officer for Parent Project Muscular Dystrophy.
Professor of Theory and Ethics in the Biosciences - University of Lübeck
Christoph Rehmann-Sutter is a philosopher, Professor of Theory and Ethics in the Biosciences at the University of Lübeck in Germany and currently also Visiting Professor at King’s College in London. Born in 1959 he obtained a first training in molecular biology with a Diploma from the Biocenter at University of Basel in 1984, then a second full training in philosophy and sociology with a doctoral degree at the Technical University of Darmstadt 1995. 1997-1998 he joined the Department of Environmental Science, Policy and Management ESPM of the University of California at Berkeley as a Research Fellow. 1996-2009 he was Head of the Unit for Ethics in the Biosciences at the University of Basel in Switzerland. 2001-2009 he was Chair of the Swiss National Advisory Commission on Biomedical Ethics, elected by the Swiss Government, advising parliament and government.
Professor of Pharmacology and Head of Laboratory of Pharmacology - University of Geneva
After twelve years of drug research in the Basel pharmaceutical industry, Urs joined the University of Lausanne as Professor of Pharmacology in 1992. Since 2004 he has headed the Laboratory of Pharmacology at Geneva – Lausanne School of Pharmaceutical Sciences, University of Geneva. Since 1992, he has had full responsibility for the entire curriculum in pharmacology for pharmacy students.
Co-founder and board member - Cure CMD
Anne is a practicing board certified emergency medicine physician in Los Angeles. Anne's daughter has congenital muscular dystrophy, subtype, dystroglycanopathy.
Director, Neuromuscular Service - The Royal Children's Hospital
Monique is a paediatric neurologist with a long interest into clinical research into the neuromuscular disorders of childhood. She has expertise in the design and performance of clinical trials - both into natural history and of pharmacotherapeutic agents - in muscle and nerve disorders of childhood, as well as a research interest into outcome measures (particularly neurophysiologic and functional measures).
Professor of Care Ethics - University of Boras
Professor Lars Sandman is a philosopher by training and received his PhD in practical philosophy from Gothenburg university in 2002. He is currently professor of care ethics at University of Borås, Sweden, and guest professor at the National Centre for Priority Setting in Health Care at Linköping University, Sweden.
Professor and Chair of Neurology - Upstate University Hospital
Jeremy Shefner, MD, PhD, is Chair of the Department of Neurology and Associate Director for Barrow Neurological Institute.
Professor and Chair of the Department of Physiology at Perelman School of Medicine of the University of Pennsylvania
H. Lee Sweeney, Ph.D., is the William Maul Measey Professor and Chair of the Department of Physiology at Perelman School of Medicine of the University of Pennsylvania. He is working to develop therapies for the skeletal and cardiac muscle aspects of muscular dystrophies using both small molecule and gene therapy approaches. Dr. Sweeney is the Director of a Wellstone Muscular Dystrophy Cooperative Research Center funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
Professor and Chief, Pediatric Cardiology, Executive Co-Director The Heart Institute - Cincinnati Children's Hospital
Dr. Jeffrey A. Towbin, Executive Director of the Heart Institute and Professor & Chief, Pediatric Cardiology at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine, did his Pediatric Residency at Children’s Hospital Medical Center in Cincinnati.
MD - University of Essen
Ulrike Schara is a paediatric neurologist at the university hospital in Essen, Germany. She has a long-standing clinical interest in muscle disorders and CMS. She has recently started to build a patient registry for CMS in collaboration with TREAT-NMD.
Head of Neuromuscular Section, Department of Neurology - Leiden University Medical Center
Jan Verschuuren is a neurologist and professor of Neuromuscular Disease at the LUMC. He is the clinical representative for the LUMC in TREAT-NMD, and president of the Dutch Neuromuscular Centre (www.ISNO.nl).
Associate Professor of Neurology - Friedrich-Baur Institut, Ludwig-Maximilians University Munich
Maggie Walter is Associate Professor of Neurology at the Ludwig-Maximilians-University of Munich. She has trained as a neurologist at the LMU Munich, and is working at the Friedrich-Baur-Institute, the neuromuscular department of the LMU, in leading position.
Independent consultant - based in the USA
In Meg’s former capacity as Director, Discovery Research at the Spinal Muscular Atrophy Foundation she has driven and overseen the Foundation’s efforts in the development and execution of new cell-based assays and drug screens and implementation of standardized drug testing in a mouse model of SMA.
Professor Dame Pam Shaw is Professor of Neurology at the University of Sheffield and Director of the Sheffield Institute for Translational Neuroscience (SITraN)
Professor Dame Pamela Shaw is Professor of Neurology at the University of Sheffield and Director of the Sheffield Institute for Translational Neuroscience (SITraN), one of the world’s leading centres for ALS/MND research.
Consultant for - SciLucent, LLC.
Tracey Zoetis, M.S. advises sponsors on regulatory and pharmacology/toxicology issues for FDA-regulated products, including pharmaceuticals, biologics, medical devices, and combination products. Her more than 25 years of experience in toxicology has afforded her a broad perspective on varied and interesting product safety issues. She has been employed by two major CROs, the FDA, and both large and small consulting firms.
Professor Neuromuscular Diseases Group - The Royal Veterinary College
After several years in general practice he moved to the USA and did his PhD on hummingbird flight. After that he returned to the UK with a lecturer post at the Royal Veterinary College where he first became involved in DMD research. From 1995 he has been based at the Imperial College.
Professor of Neuromuscular Genetics - Newcastle University
Prof. Kate Bushby MD FRCP is a Professor of Neuromuscular Genetics. She is a clinical academic with joint appointments between Newcastle University and the NHS. The Muscle Team within the Institute of Genetic Medicine is a partner in the MRC Centre for Neuromuscular Diseases, of which Prof Bushby is Deputy Director.
Professor of Neuromuscular Genetics - Newcastle University
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.
TREAT-NMD Programme Coordinator
Becca Leary is a member of the TREAT-NMD Secritariat and responsible for the Global SMA and DMD registries.
DMD Programme and TACT Coordinator - Newcastle University
Cathy Turner is Duchenne Programme and TACT Coordinator at the John Walton Muscular Dystrophy Research Centre. Cathy has also been responsible for supporting the NeurOmics project with significant involvement in communications, data-sharing and ethics. Prior to this she was the communications officer for BIO-NMD. In addition, Cathy works closely with patient organisations on improving patient and family involvement in neuromuscular research and is part of a small team currently developing interactive digital tools to support children taking part in paediatric clinical trials.
Cathy has a background in journal publishing with Elsevier Science and Emerald Insight and then worked as a science teacher for 5 years before joining the team here at Newcastle University. She has a degree in biochemistry, a post-graduate teaching certificate in science and a Masters in Business Administration.
Web Development Officer - Newcastle University
Michael joined the TREAT-NMD team in March 2009 and is responsible for maintaining and developing the TREAT-NMD website.