The TREAT-NMD Advisory Committee for Therapeutics (TACT) recently hosted its first review meeting in Rome, Italy. Cristina Csimma (Virdante Pharmaceuticals Inc), as TACT chair, is leading this exciting initiative with the support of the TACT core, nominated committee members and the TREAT-NMD secretariat. TREAT-NMD is confident that the breadth and calibre of the international TACT experts will ensure the quality of the output.

The aim of TACT is to provide transparent and consistent guidance and advice to the neuromuscular community, in an educational and directional context, on the readiness of drugs and/or therapeutic targets for trials in neuromuscular diseases.

The remit of the committee is to evaluate the therapeutic potential of drugs based on preclinical data, thereby assisting TREAT-NMD in prioritising clinical trials to be run via the network. TACT advice will be helpful for preparing funding applications and investigational drug applications, and serve as an unbiased appraisal to be published for the wider neuromuscular community. We hope that this process would be endorsed by the major funders.

In addition to the expert analysis of preclinical data TACT will also address issues of drug formulation, bioavailability and toxicology as well as possible regulatory requirements and marketing considerations. In close collaboration with the TREAT-NMD clinical trials coordination centre in Freiburg TACT is also dedicated to providing information on rational and economical trial design.  The ultimate goal of TACT is to help pave the pathway for successful drug registration by providing professional and independent advice.

The TACT appraisal process of potential therapeutics for clinical trials will take a maximum of 6 months. TACT will consider more than one drug at a time and therefore will have panels of experts, selected from the TACT members below, reviewing the drugs concurrently, with the same chair and core committee for continuity.

TACT are currently accepting enquiries for their second round of applications please contact to express your interest. The next TACT review meeting is scheduled for 5th & 6th June 2010.
 

TACT Application Form

A sample version of the TREAT-NMD Advisory Committee for Therapeutics Application form is now available to download here.

This sample form is meant to be used for reference purposes only and is a static version of the online form that is used to submit applications.

If you wish to submit an application please contact for details of the online form's URL.

Download the sample application form here.
 
Download Terms of reference document here

Core Committee Members

Vice President, Drug Development, Virdante Pharmaceuticals Inc, Cambridge, MA, USA.

Cristina currently leads the drug development activities for Virdante Pharmaceuticals, a biotechnology company based in Cambridge, US, before which she was a Principal at Clarus Ventures, a premier life sciences venture capital firm investing in biopharmaceuticals and medical technology.  She has 20 years of global drug development experience in the biotechnology, pharmaceutical and academia sectors.

Prior to joining Clarus Ventures, Dr. Csimma was Senior Director of Translational Research at Wyeth Research. In that capacity she oversaw a broad range of exploratory clinical studies, including in muscular dystrophy, and led the integration of biomarkers and novel technologies into clinical development. Prior to Wyeth, Dr. Csimma held leadership clinical responsibilities at Genetics Institute, including the global development and approval of rhBMP-2, the first ortho-biologic approved for fracture healing. Her experience encompasses multiple therapeutic areas and global Regulatory interactions.

Prior to joining the pharmaceutical industry, she was a clinical pharmacist at the Dana Farber Cancer Institute. In the past few years Dr. Csimma has been a member of MDA’s Translational Research Advisory Committee and NIH NINDS’ Advisory Panel for Translational Research and she is currently a member of the MDA’s Venture Philanthropy Scientific Advisory Board.

Head of the Regulatory Affairs Department at GENETHON, France.

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. He is responsible for communication with national agencies (Afssaps; MHRA; PEI) the EMEA and the European commission (DG Enterprise).

For AFM he has been responsible for successfully preparing and obtaining Clinical Trial Authorisation in France for products which are already on the market in another clinical indication. Previously, he has worked in Regulatory Affairs, mainly on chemical molecules.
 

Assistant Professor of Neurology, Columbia University, USA.

Petra Kaufmann, M.D., M.Sc., is Director of the Office of Clinical Research (OCR) at the National Institute of Neurological Disorders and Stroke (NINDS). 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.

Prior to joining NINDS, Dr. Kaufmann was a tenured associate professor of neurology at Columbia University in New York City.  A native of Germany, she earned her medical degree from the University of Bonn, Germany, and a master of science degree in biostatistics from Columbia’s Mailman School of Public Health. She completed an internship in medicine at St. Luke’s/Roosevelt Hospital in New York City, and trained in neurology and clinical neurophysiology at Columbia University.   Dr. Kaufmann is board-certified in neurology, clinical neurophysiology, neuromuscular medicine, and electrodiagnostic medicine.

She did a postdoctoral fellowship in molecular biology of mitochondrial diseases at Columbia’s H. Houston Merritt Center for Muscular Dystrophies and Related Diseases.  While on the faculty of Columbia University,  she worked clinically in the neuromuscular division, the electromyography laboratories, and the pediatric neuromuscular clinic.  Her research focused on the clinical investigation of spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS) and mitochondrial diseases.

Head of the Department of Neuropediatrics and Muscular Disorders at Children’s Hospital, University Hospital 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 has held his current position since 1990. He has published on a broad range of topics in child neurology, and during the last 18 years has concentrated on clinical scientific work in neuromuscular disorders. He has conducted several German multicentric trials in NMD as principal investigator, and he is head of the Trials Organisation Center of MD-Net and The Clinical Trials Coordination Center of TREAT-NMD.

Senior Consultant, PharmaMacLLC, USA.

John’s expertise is drug discovery, risk minimization, and medicinal chemistry. He has worked in a number of diseases including different acute and chronic neurologic disorders.  He is currently President, PharMac LLC.  He began his career as a medicinal chemist with Upjohn and has subsequently held positions with Pharmacia and Upjohn, Pharmacia, and Pfizer.  John was Director of CNS Research with Upjohn, Vice President and Global Head of Chemistry for both Pharmacia and Pharmacia & Upjohn and Vice President Research with Pfizer.

John currently chairs a NINDS development team in the spinal muscular atrophy area, serves on administrative and scientific advisory boards, consults, participates as a NIH study section member, works with three venture capital funds, and is a partner with the Apjohn Group. His Board and Scientific Advisory Board positions include Psychogenics, UCB, CeeTox, Avaant, Deciphera, Michigan High Throughput Screening Center, Verseon, CINRG, Reaction Biology, Scripps, AdmetRx, and PharmOptima, John holds 53 US patents and has over 60 refereed publications. 

Medical Officer, Food and Drug Administration, Rockville Maryland, USA.

Elizabeth has been a medical officer at the US Food and Drug Administration (FDA) since 2002.  She is a board-certified pediatric neurologist and was a primary reviewer and then team leader for neuromuscular diseases in the neurology review division at the FDA before joining the Office of Orphan Product Development. While at the FDA, her review experience has included primary reviews for orphan drug designation reviews, New Drug Applications (NDA)/efficacy supplement reviews, phase III protocol reviews (including special protocol assessments) and numerous commercial/non-commercial Investigational New Drug (IND) reviews.

She has 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 has worked to develop new scientific, clinical and regulatory standards for the development of certain drug classes. Her initiatives have 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.

Director, Center for Gene Therapy, Columbus Children’s Research Institute, Professor of Pediatrics and Neurology, Ohio State, University, USA.

Jerry is the Director of the Centre for Gene Therapy and a Professor of Pediatrics and Neurology, with significant experience. His areas of interest include translational laboratory studies related to gene therapy of muscle disease,  clinical trials in neuromuscular disease (gene replacement, gene restoration, pharmacologic manipulation) and Clinical characterization (genotype - phenotype correlations) in neuromuscular disease.

Associate Professor of Pediatrics, Children's National Medical Center, Washington DC, USA.

Raju is an associate professor of pediatrics with a robust translational research program in muscular dystrophy. He currently direct a preclinical drug testing facility (MPDTF)  dedicated for neuromuscular disorders at the Research Center for Genetic Medicine, Children’s National Medical Center which has grown to be the largest academic facility in the US for muscular dystrophies. During the last two years the facility has taken a lead in collaboration with TREAT-NMD and helped to draft and develop consensus standard operating procedures (SOPs) for preclinical end points for muscular dystrophy models. The facility has so for conducted 40 different preclinical trials in four different models of muscular dystrophy. Raju also works closely with CINRG clinical trial network team in to identify therapeutic targets that are ready for human clinical trials in DMD.

Head of Department of Cellular and Molecular Neuroscience, Imperial College, London, UK.

Dominic has been involved with neuromuscular diseases since 1990 and has extensive experience working with transgenic mice and other laboratory animals as well as having a practicing veterinary background. He has been involved with a number of workshops examining welfare and best practice in animal research as well as the TREAT-NMD standardisation efforts for analysis of results in the mdx mouse. Therefore he has a good understanding of the role and limitations of pre-clinical models for a range of conditions. He was also involved in putting together the pre-clinical package and other aspects of the regulatory paperwork for the first antisense clinical trial run by the MDEX consortium and liaising with the UK MHR and GTAC.
 

Extended Committee Members

Associate Professor of Pediatrics & Child Neurology, School of Medicine, Leuven  University, Belgium.

Gunnar Buyse, MD PhD, is Associate Professor of Pediatrics and Child Neurology at the School of Medicine of the University in Leuven, Belgium (Katholieke Universiteit Leuven), and is deputy clinical chair at the division of Child Neurology of the University Hospitals of Leuven.

His clinical and research interests cover the entire spectrum of child neurology. Gunnar Buyse has extensive experience in basic, translational and clinical research. He holds a PhD in basic medical sciences (molecular biology and biophysics). His group has contributed to preclinical phases of drug development, by doing very long-term randomized controlled therapeutic preclinical studies in animal models of muscular dystrophies.

He has been principal or co-investigator of several clinical randomised controlled trials (including international multicentric) in the field of pediatric neuromuscular diseases. As such he has the unique expertise of bringing basic sciences first to the preclinical stage and then to gold-standard investigational human clinical trials.

Professor, Department of Pharmacobiology, University of Bari, Italy.

Annamaria has a long lasting experience in preclinical research on pharmacological treatments for DMD and collaborates with leading scientists in the field. She developed assay conditions for mdx and is familiar with many methods used to test drug efficacy, especially muscle functionality and electrophysiology. She has a profound knowledge of mouse physiology and pathology and hence of the critical points that make out the quality of a study. She is actively engaged in the pre-clinical work within TREAT-NMD and started working at the harmonization of preclinical studies on mdx before TREAT-NMD was launched.

Professor, The University of Western Australia, Crawley, Australia.

Miranda has devoted over 30 years of research to better understanding the process of damage and repair of normal skeletal muscle.  This extends into trying to more precisely define the early events downstream of the dystrophin deficiency that are responsible for the pathology of DMD. She has a long standing interest in cell therapy to replace the defective gene and more recently in pharmaceutical approaches to reduce the severity of the disease.

Her research has a strong engagement with DMD Parent Groups (in the USA, Europe, UK, Brazil) and she is on the board of the Norwegian Parent Project Advisory Committee.  She recently initiated an Indian Ocean Rim Muscle Colloquium (2009) to facilitate networks in this region.

Director, Research Center for Genetic Medicine & The Wicker Project for Muscular Dystrophy Research, Children's National Medical Center.

Eric is the Scientific director of CINRG, and has extensive experience with pre-clinical and clinical drug development. In 1990, he established an independent laboratory where he worked on muscular dystrophies, ion channel disorders, and other neurological conditions.  In 1999, he established the Research Center for Genetic Medicine Research at Children’s National Medical Center.

He is experienced in most aspects of human molecular genetics, with NIH funding in SNP association studies, microarrays in human disease, and management of large public access databases. He is board-certified by the American Board of Medical Genetics in Clinical Molecular Genetics and ran the University of Pittsburgh Clinical Molecular Genetic training program.

He has a strong commitment and track record as a molecular biologist and a teacher and has extensive experience in administering large and complex research teams.  Eric is among the most highly cited scientists, as tracked by ISI Inc, Philadelphia PA. 

Research Director, Families of SMA, USA.

Jill has significant industry expertise is in pre-clinical drug development.  She has led project teams that have developed HTS assays, conducted high-throughput screens, and validated compounds in relevant cell-based assays.  In this capacity she has worked on a SMA drug program, as well as several cancer-focused projects. 
 
For the past 4 years, she has been the research director at Families of SMA and oversees all of Families of SMA research investments.  From this experience she has developed a clear understanding of the basic research arena in SMA and what currently constitutes the SMA therapeutics pipeline.  

Lecturer, Université Catholique de Louvain, Belgium.

Jean-Marc is a motivated and excellent scientist, he holds an interesting position between clinics and research and his experience covers DMD and SMA. He works with leading scientists in the preclinical research of both dieseases, has a profound knowledge of the animal models and of many functional and anatomical assays. He is a keen collaborator in TREAT-NMD and is precise in the evaluation of scientific assessments and data. 

Director, Discovery Research Member, Spinal Muscular Atrophy Foundation, USA.

In Meg’s 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. In addition she has worked closely with the mouse transgenics team at Regeneron, Inc, in creating new allelic series and conditional rescue mouse models for SMA, which are now being characterized.

Through the SMA Foundation grants program she has worked with multiple investigators in both testing therapeutic candidates and in developing specific new mouse strains for testing therapeutic hypotheses.  She has had extensive exposure to a wide variety of cellular and animal models, plus small molecule and biologic therapeutic candidates, for SMA and related conditions. 

Pharmaceutical Research and Development Executive, Mike Pleiss and Associates, LLC, USA.

Mike has been working in the field of neurodegeneration since 1992 when he joined Athena Neurosciences (now Elan). He 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.  He was VP of global chemistry and drug metabolism & pharmacokinetics (DMPK) and supervised over 60 FTEs. He played critical roles in all aspects of drug design and development through Phase I.

Mike left Elan in September 2005 and began consulting in all aspects of drug design and development. The majority of his clients have been foundations, many working in the neurodegeneration field including the SMA Foundation and the Myelin Repair Foundation (MRF). Mike serves as the chairman of the MRF Drug Discovery Advisory Group whose efforts mirror those of the TACT committee. He is  also working closely with several research groups at the Gladstone Institute on novel approaches for AD, MS and Huntington’s disease, the majority of this effort is directed at repositioning of current therapeutics for these indications.

Professor of Pharmacology and head of Laboratory of Pharmacology, University of Geneva, Geneva.

Since 2004 Urs has held the position of Professor of Pharmacology and head of Laboratory of Pharmacology at Geneva – Lausanne School of Pharmaceutical Sciences, University of Geneva. In this position he has full responsibility for the entire curriculum in pharmacology for pharmacy students.

His recent research interests include pharmacology of cellular calcium regeneration and with his research team, he focus on altered calcium handling in Duchenne muscular dystrophy & pharmacological approaches.
Urs is also a member of the Conseil Scientifique de l’Association Française contre les myopathies and an expert panelist of the Swiss National Science Foundation.
 

Senior Director, Therapeutics Programmes, Summit plc. UK.

Jon is the Senior Director for the Therapeutics Programmes in Summit PLC and a TREAT-NMD partner. He has direct experience with the aspects necessary to identify potential compounds for triage into trials. During his time at Summit(previously VASTox) he has been involved in taking both DMD and SMA projects from this early scientific due diligence through to conceptual clinical trial design with regulator friendly endpoints. He has good working relationships with the various patient organisations in Europe and the US and with key personnel within the orphan disease groups of both the EMEA and the FDA.

Director, Multi-Center Studies Section, Center for Clinical and Community Research, Children’s National Medical Centre.

Avital is a Statistician with extensive experience in multi-site trial design, data management, and data interpretation.  She joined the Children’s National Medical Center (CNMC) in May, 2008 as Director, Multi-Center Studies Section in CNMC’s Children’s Research Institute. Prior to joining CINRG she was Professor of Biostatistics in Pediatrics at the University of Pennsylvania School of Medicine, as well as Chief of the Division of Biostatistics and Epidemiology at the Children’s Hospital of Philadelphia (CHOP).

She continues to hold an adjunct professor appointment at the University of Pennsylvania. Avital has over 20 years experience in clinical research and multi-center clinical trials. She is the PI of the Coordinating Center for The Childhood Absence Epilepsy Study. As a statistician, her interests are in survival analysis, longitudinal analysis methods, and issues in design and analysis of clinical trials, such as compliance and site effects. She is an author on more than 120 peer-reviewed publications and a Fellow of the American Statistical Association.

Senior statistician, and Scientist at University Medical Center, Freiburg.

Dieter is currently a Scientist at the Institute for Medical Biometry and Medical Informatics, University Medical Center Freiburg. Previously he has held the position of Head of the department of Biometry and Head of the Department of Preclinical Biometry. His particular research interests are in theoretical aspects of equivalence trials and its application to pharmaceutical drug development including bioequivalence, non-inferiority and “proof of safety“. He has been an invited speaker at various workshops of the German Region of the International Biometric Society and the Drug Information Association. He is the author or co-author of more than 70 research articles and book-chapters and co-author of the textbook “Bioequivalence in Drug Development: Methods and Applications, Wiley, Statistics in Practice“.
 

Director Neurology Residency Program, University of Padova, Italy.

Corrado is Director of the Neurology Residency Program at the University of Padova. He has coordinated numerous trials in the use of steroids in DMD and participated in the SMART trial in spinal muscular atrophies, sponsored by Telethon. He is an expert on metabolic myopathies and coordinates a group of physicians involved in ERT in adult glycogenosis type II.

Director, Office of Investigational Therapeutics, Children’s National Medical Center, Washington, USA.

As previous Chief Medical Officer of MedImmune (1993-2008) Ed has extensive experience in clinical trials (maternal AIDS transmission) and drug development (Synergis, Flumist, others), including shepherding of complex protocols through FDA.  He has joined Children's National Medical Center recently as Director in the Office of Investigational Therapeutics and is up to speed on DMD clinical trial development; he is an outstanding resource for the neuromuscular community.
 

Director, Neuromuscular Program, Childrens Hospital Philadelphia,  PA, USA.

Richard is a paediatric neurologist  and director of the neuromuscular programme at the Children’s hospital of Philadelphia. His special interests include Neuromuscular Disorders, pediatric electromyography and metabolic disorders of the nervous system. His research interests are concerned with the development of outcome measures and with the design and execution of clinical trials in spinal muscular atrophy, Duchenne muscular dystrophy, Fabry and Pompe diseases. 
 

Chief, Neurogenetics Branch, NINDS, NIH, Bethesda, MD, USA.

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 dystrophy, spinal muscular atrophy, Friedreich's ataxia, and SBMA. He serves on advisory boards for voluntary organizations that are sponsoring similar therapeutics development initiatives, including the SMA Foundation, the ALS Association, and MDA Venture Philanthropy. He is also on the Data and Safety Monitoring Board for CINRG. He has experience with industrial therapeutics development from a sabbatical at Merck in 2005 and from subsequent service on advisory boards for Merck and Biogen Idec.

Adjunct Associate Professor, University of Utah School of Medicine, Department of Pediatrics, Salt Lake City, UT, USA.

Kevin is the Director of the United Dystrophinopathy Project, a NIH-funded consortium studying the natural history and genotype/phenotype correlations in the DMD gene.  He has participated in several recent clinical treatment trials, including use of MYO-029 (Wyeth, Inc.), IPLEX (Insmed, Inc.), and PTC124 (both 2a and 2b studies; PTC, Inc.).  He has served on expert advisory committees for developing clinical protocols for exon skipping therapies, and for other therapies in pre-clinical development. 

Consultant Neurologist at Pitié-Salpêtrière Hospital, France.

Pascal’s field of expertise includes adult neuromuscular disorders. He has been involved in several clinical trials in the previous years and has a wealth of experience: Salbutamol in FSHD (principal investigator), DHEA in myotonic dystrophy (co-investigator), exercise training in McArdle’s disease (co-investigator), myoblast graft in OPMD (co-investigator), Bezafibrate in CPTII deficiency (co-investigator), Enzyme replacement therapy in late-onset Pompe disease (PI).

Paediatric Neurologist, The Royal Children’s Hospital, Victoria, Australia.

Monique is a clinician in pediatric neurology with a long interest in 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). She has for some years been a PI for the CINRG study network, firstly for Sydney and now for Melbourne, Australia, and she is the Melbourne PI for the PTC124 trial. She has also recently completed the first paediatric trial of vitamin C for CMT type 1A, which is now in press. She has good collaborative relationships with other neuromuscular centres in Australasia.

Director of Cardiology and Co-Director of Heart Institute at Cincinnati Children's Hospital.

Jeffrey is the Director of Cardiology and Co-Director of Heart Institute at Cincinnati Children's Hospital, before which he was chief of Pediatric Cardiology at Texas Children's Hospital. Jeff has active clinical and research interests. His major research areas of interest focused on cardiomyopathies, myocarditis, and other causes of sudden cardiac death and congenital heart disease. Clinically, he focuses on diagnostic and therapeutic advances in cardiomyopathy and transplant care and Clinical Cardiac Genetics.
 

Head of Neuromuscular Section, Department of Neurology, Leiden University Medical Center.

Jan is actively involved in the therapeutic trials going on in the field of Duchenne muscular dystrophy. In his clinical work as a neurologist he has been principal investigator or otherwise participated in several trials and clinical investigations, in DMD but also in other neuromuscular diseases.

Professor, Academic Medical Centre, Department of Neurology,  Amsterdam.

Marianne has been the leader of the neuromuscular group at the Academic Medical Centre in Amsterdam, The Netherlands for more than 20 years. During this time her group has contributed to research on muscular dystrophies, motor neuron diseases and hereditary neuropathies.

Professor of Pediatrics, Scientific and Medical Director, Institut de Myologie, France.

Thomas currently holds the positions of Professor of Pediatrics (Professeurs des Universités –Praticien Hospitalier), Université Pierre et Marie Curie Paris VI, Scientific and Medical Director, Institut de Myologie, Groupe Hospitalier de la Pitié-Salpêtrière, Paris and Director, Mixed Research Unit UMR_S 974 UPMC-INSERM-CNRS-AIM.

He is one of the leaders in the neuromuscular field with over 188 articles in MEDLINE and 20 book chapters.

Thomas was and is a member of several scientific committees including the Muscular Dystrophy Campaign; Clinical Adviser for Conseil Scientifique, Association Française contre les Myopathies; Institut des Maladies Rares; MRC Centre for Neuromuscular Disease; Dutch Duchenne Parents Project and Integrated Research Centres, German Ministry of Education and Research.  He is also a member of the Executive Board for the World Muscle Society .

Associate Professor of Neurology, Department of Neurology, The Johns Hopkins Hospital. Baltimore.

Kathryn is an Associate Professor of Neurology at Johns Hopkins. Her work focuses on neuromuscular and neurogenetic diseases with a special emphasis on hereditary muscle diseases. She cares for patients with a variety of disorders, including Duchenne muscular dystrophy, Becker muscular dystrophy, FSH dystrophy, Myotonic muscular dystrophy and Limb Girdle muscular dystrophy.

Kathryn is co-director of an NIH-funded, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center focusing on modulation of muscle growth for muscular dystrophies. Her research focuses on mechanisms to promote muscle regeneration for a variety of neuromuscular disorders. She is the principal investigator in the multicenter trial of an inhibitor of myostatin in adult muscular dystrophy. Other clinical trials aimed at enhancing muscle growth or decreasing muscle degeneration are also being conducted by her clinical research group.
 

Assistant Professor for Neurology, Friedrich-Baur-Institute, Munich, Germany.

Maggie is an Assistant Professor for Neurology and a TREAT-NMD Partner within MD-NET (which she co-coordinates). She has broad experience in planning, conducting, analyzing and publishing clinical studies. Therefore she can advise on practicalities of recruitment to a particular protocol, and on other general protocol issues.

Director Pediatric Neuromuscular Program and MDA Clinic, Division of Neurology, Cincinnati Children’s Hospital Medical Center, USA.

Brenda is the Director of the Pediatric Neuromuscular Programme and MDA clinic.  She has not only been active in the care and management of pediatric patients with neuromuscular disorders, but has also actively participated in clinical trials for DMD and in steering committees for the PTC 124 study trials and neuromuscular clinical trial outcomes.

Consultant Physiotherapist - PhD MSc MCSP

Michelle initially specialised in Rheumatology and gained a masters degree in the clinical evaluation and development of outcome measures in Ankylosing Spondylitis. Since 1990 she has worked for the Newcastle Muscle centre where she is a consultant physiotherapist. Michelle has published on the management of various NMDs and her PhD in 1996 evaluated the impact of nocturnal ventilation on survival in DMD.

Michelle is a consultant for various pharmaceutical companies specifically relating to clinical evaluation including Wyeth, Santhera, PTC, Prosensa and AVI. She also has wide experience in the development of operational manuals and of training clinical evaluators for clinical trials.
 

CEO, Parent Project Muscular Dystrophy (PPMD), US.

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.  

Along with leading PPMD, Pat speaks about Duchenne and related topics at conferences each year worldwide and is an active Board member with the Genetic Alliance and the Muscular Dystrophy Coordinating Committee, U.S. Department of Health & Human Services. She is also a committee member on the Collaboration in Education and Test Translation Program; and serves on the data safety monitoring board for both the Rare Diseases Clinical Research Network and Cooperative International Neuromuscular Research Group and TREAT-NMD STAC.
 

Board of Directors, AFM, France.

Marie-Christine is 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.
 

Professor of Theory and Ethics in the Biosciences at the University of Lübeck in Germany.

Christoph is experienced in the field of bioethics and is a member of the TREAT-NMD Project Ethics Council. He has served as a member of a research ethics committee (EKBB) in Basel for 9 years, which evaluates approximately 350 clinical research studies per year. Since 2001 he has been the Chair of the Swiss National Advisory Commission on Biomedical Ethics.

CEO, Action Duchenne.

Nick is the CEO of Action Duchenne a UK non profit that funds research for new medicines for Duchenne and campaigns to improve access to better medical care. He has an honours degree in Chemistry and a specialist diploma in teaching children with specific learning difficulties. Nick has a son Saul who is 9 who has Duchenne.
 

Board of Directors, AFM, France.

Béatrice is a mother of a boy affected by DMD. She is a member of AFM Board of Directors and the coordinator of AFM International Committee.
 

Director of Research, Muscular Dystrophy Campaign (MDC), UK.

Marita is the Director of Research at the Muscular Dystrophy Campaign which has funded high quality research into neuromuscular disorders for the last 50 years.  She in charge of an annual £2m budget invested to increase understanding of muscle function and muscle disease and to ensure an efficient bench-to-bedside transition of promising technology.   

In her role, Marita is close to affected individuals and their families with a range of muscular dystrophies and related neuromuscular conditions and understands their “sense of urgency” to find treatments. She believes that research is now entering an exiting phase and that it is vital that patient organisations work together with the scientific community to have an input in the prioritisation and development of therapeutic approaches.

Marita has also worked as a scientist with a speciality “Molecular Genetics” for more than 15 years and was for some time involved in developing a gene therapy approach for Duchenne muscular dystrophy. She can appropriately communicate the advances and progress to patients and their families.
 

Chair, United Parent Project Muscular Dystrophy (UPPMD), The Netherlands / Europe.


Elizabeth is the president of Dutch Duchenne Parent Project and the chair of United Parent Projects Muscular Dystrophy (UPPMD). UPPMD is owned and managed by parent project organisations set up by parents of children with Duchenne Muscular Dystrophy in many different countries all over the world. As the chair of UPPMD, Elizabeth can represent the Duchenne parent community on the TACT committee.

Elizabeth is invited to speak at many international conferences about Duchenne. She is also a member of the TREAT-NMD Project Ethics Council and a practicing Orthodontist.  
 

Secretariat

Professor of Neuromuscular Genetics

Professor Kate Bushby has overseen the expansion of the Newcastle Muscle Centre since 1999 to its position today as a leading international neuromuscular centre. Together with Volker Straub, Katie is joint coordinator of the TREAT-NMD network.

She is Research Director of the ENMC and is a member of the Scientific Advisory Committee of the AFM. In the UK she is Deputy Director of the MRC Centre for Neuromuscular Diseases at UCL and Newcastle and Clinical Director of the NCG diagnostic and advisory service for rare neuromuscular disorders (limb-girdle muscular dystrophies).

TREAT-NMD Assistant Project Manager

Emma joined the TREAT-NMD network in December 2006 and is responsible for leading the work relating to shared activities and tools; developing strategies for extension of the network activities into other disease groups; enhancing international relations and collaborations; and developing and improving access to comprehensive training and education activities. Her background is in Health and Human Sciences and Biological Anthropology.  For six years she worked as a Project Coordinator in the Durham University Sleep Lab studying mother-infant sleep. During this time she completed her Biological Anthropology MSc (Research).

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.  Together with Hanns Lochmüller, Volker was responsible for setting up the German muscular dystrophy network, MD-NET, of which he was joint coordinator until 2008. Within the neuromuscular research group at Newcastle, Volker has a long-standing interest in the pathogenesis of muscular dystrophies, with research using zebrafish and mouse models.  His current research also involves the application of contrast enhanced MRI.