Global database oversight committee - TGDOC

The TREAT-NMD Global Database Oversight Committee (TGDOC) is composed of representatives of the TREAT-NMD network plus representatives of patient organisations and each national registry. The Chair of the committee is Dr Hugh Dawkins and the Chair-elect is Dr Nathalie Goemans.

The TGDOC is responsible for reviewing all requests for data from the global database. This is intended to be a streamlined and rapid procedure in order not to delay approval. Requests will be responded to within a set time period of two weeks.

A list of all committee members is provided below, together with their disclosure statements.

Explanatory note: Examples of financial interest/arrangements to be disclosed include employment, grant support, honoraria, equity, ownership and royalties from a commercial organization within the last 3 years (after 01-01-2009) and valued at more than 2,000 Euro.

 

Nathalie Goemans Nathalie Goemans - Chair of TGDOC

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.

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Craig Campbell Craig Campbell - Vice Chair - TGDOC

Pediatric Neurologist - London Health Sciences Centre

Dr Campbell practices at the Children’s Hospital-London Health Sciences Centre and at the University of Western Ontario, where he is an Associate Professor of Pediatrics, Clinical Neurology, and Epidemiology and Biostatistics. He is currently the medical director of the Pediatric Neuromuscular Clinic and the Pediatric Neurophysiology Laboratory.

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Hugh Dawkins Hugh Dawkins - Outgoung Chair of TGDOC

Office of Population Health Genomics, Public Health Division, Department of Health
Curator of Australian National DMD Registry

Professor Dawkins leads the OPHG in the development and management of policies, plans and statewide services to minimise the impact of rare diseases. He provides advice and recommendations on issues relating to genetic testing, screening, gene patents, genetic privacy, stem cell therapy and the translation of new knowledge and technologies to improve in health services.

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Anna Ambrosini Anna Ambrosini

Research Program Manager - Fondazione Telethon
Representative of the Italian registry of patients with neuromuscular diseases-onlus

Anna Ambrosini (PhD in Pharmacology and Toxicology) has 15 years experience in research laboratories active in the field of neuroscience. Since 2001 she has been Research Program Manager for the Italian Fondazione Telethon, where, in particular, she is responsible for developing programs in the field of inherited neuromuscular diseases.

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Svetlana Artemieva Svetlana Artemieva

Paediatric Neurologist - Moscow Region Paediatric Psychoneurological Hospital
Curator of Russian (Moscow) DMD and SMA Registries

Dr Svetlana Artemieva is head of the paediatric neurology section for the Moscow region at the Moscow Region Paediatric Psychoneurological Hospital No. 1. She is also responsible for the Russian patient registry for DMD and SMA together with the patient organization Nadezhda.

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Alexander N. Baranov Alexander N. Baranov

Senior Scientist, Head of Gene Therapy Group - Russian Academy of Medical Sciences
Curator of Russian (St Petersberg) DMD and SMA Registries

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Farhad  Bayat Farhad Bayat

Head of Laboratory at the Pasteur Institute of Iran
Curator of the Iranian DMD Registry

Farhad Bayat, MS, works as a Head of Laboratory at the Pasteur Institute of Iran. He is a patient organisation representative. His background is in organic chemistry and in quality control of recombinant proteins & vaccines.His main topics of research are new drug delivery systems and nanomedicines.

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Christophe Béroud Christophe Béroud

Assistant Professor - INSERM
TREAT-NMD work package leader for patient registries

Christophe Béroud (PharmD, PhD): has 22 years experience in research laboratories and is an expert in bioinformatics (LSDBs) and molecular biology (diagnostic of DMC).

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Ria Broekgaarden Ria Broekgaarden

Dutch Patient Organisation Representative - Vereniging Spierziekten Nederland - VSN
Patient organisation representative

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Filippo Buccella Filippo Buccella

Italian DMD Registry Curator - Parent Project - Genitori contro la Distrofia Musculare di Duchenne & Becker ONLUS Italy
Curator of Italian DMD/BMD Registry

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.

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Rasha El Sherif Rasha El Sherif

Consultant of Neurology and Head of Neurogenetics Unit - Air Hospital, Egypt
Curator of the Egyptian DMD registry

Dr Rasha El Sherif is a Consultant of Neurology and a Head of Neurogenetics Unit at the Egypt Air Hospital. She is also a researcher in the laboratory of neurogenetics at the Ain Shams University. Dr EL Sherif underwent training on molecular diagnosis of muscle disease (genetic diagnosis of DMD) at the National Institute of Neuroscience NCNP Tokyo, Japan in 1998, 2000, 2008 and 2010, where she was a visiting researcher in 1998 and 2000. She holds a PhD in neurology obtained from the Ain Shams University in 2010.

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Kevin Flanigan Kevin Flanigan

Professor of Pediatrics and Neurology - Ohio State University College of Medicine
Director of the United Dystrophinopathy Project (UDP) Registry

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.

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Ole Gredal Ole Gredal

Curator of the Danish TREAT-NMD DMD and SMA registry - The Rehabilitation Centre for Neuromuscular Diseases
Curator of the Danish DMD and SMA Registries

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Serap İnal Serap İnal

Turkish DMD and SMA Registry Curator (Istanbul) - Istanbul University
Curator of Turkish (Istanbul) DMD and SMA Registries

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Jacqueline Jackson Jacqueline Jackson

USA SMA Registry - Indiana School of Medicine
Curator of US SMA Registry

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Anna Kaminska Anna Kaminska

Polish DMA and SMA Registry - Medical University of Warsaw
Position in Global Database Oversight Committee - Coordinator of Polish DMD and SMA Registries

Anna Kaminska is Professor and Head of the Department of Neurology at the Medical University of Warsaw, Poland. She received the MD and PhD in Medical Sciences from the Medical University of Warsaw.

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A. Ayse Karaduman A. Ayse Karaduman

Turkish DMD and SMA Registry Curator (Ankara) - Hacettepe University
Position in Global Database Oversight Committee - Curator of Turkish (Ankara) DMD and SMA Registries

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Veronika Karcagi Veronika Karcagi

Head of Department of Molecular Genetics and Diagnostics - Semmelweis University
Curator of Hungarian DMD and SMA Registries

Dr. Veronika Karcagi, PhD is Head of Department of Molecular Genetics and Diagnostics at the National Institute of Environmental Health in Budapest.

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En Kimura En Kimura

Chief of Early Stage Exploratory Clinical Trial Unit - National Institute of Neuroscience - National Center of Neurology and Psychiatry
Curator of Japanese Muscular Dystrophy Registry (Remudy)

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Janbernd Kirschner Janbernd Kirschner

Consultant Pediatric Neurologist - University Medical Center Freiburg
Head of TREAT-NMD Care and Trial Site Registry (CTSR)

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

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Jaana Lähdetie Jaana Lähdetie

Senior Consultant in Child Neurology - Turku University Central Hospital
Curator of Finnish DMD and SMA Registries

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Hanns Lochmüller Hanns Lochmüller

Professor of Experimental Myology - Newcastle University
Oversight Committee Member (former chair of TGDOC)

Professor Hanns Lochmüller trained as a neurologist in Munich (Germany) and Montreal (Canada). He was appointed chair of experimental myology in the neuromuscular research group at the Institute of Genetic Medicine of Newcastle University in 2007 and was elected Chair of the TREAT-NMD Alliance Executive Committee in April 2012. Hanns is also scientific coordinator of EuroBioBank and leads the activity on “patient registries and biobanks” for TREAT-NMD.

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Vitaliy Matyushenko Vitaliy Matyushenko

President of the Children with SMA Charitable Foundation - Children with SMA
Curator of Ukranian SMA Registry

Vitaliy grew up in the Ukraine and graduated in 1993 from Kharkiv National University after specializing in nuclear physics. He worked both in this area and as a forensic investigator until the birth of his daughter. At the age of one she was diagnosed with spinal muscular atrophy and as a result of this in 2004 Vitaliy founded the Foundation “Children with SМА”.

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Violeta Mihaylova Violeta Mihaylova

Bulgarian DMD and SMA Registry - University Hospital Alexandrovska
Curator of Bulgarian DMD and SMA Registries

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Vedrana Milic-Rasic Vedrana Milic-Rasic

Curator of DMD and SMA Registry in Serbia - University of Belgrade
Curator of Serbian DMD and SMA Registries

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Lauren Morgenroth Lauren Morgenroth

Operations Manager, CINRG and Children’s National - CINRG
Curator of the CINRG Database

Lauren has been involved with the Cooperative International Neuromuscular Research Group (CINRG), an academic clinical trial network, for over 10 years. She is the central genetic counselor and operations manager for the Coordinating Center at Children’s National in Washington, DC.

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Ian Murphy Ian Murphy

Spanish DMD Registry

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Carmen Paradas Lopez Carmen Paradas Lopez

Curator of Spanish Neuromuscular Diseases Registry

Carmen Paradas Lopez, PhD, MD is a Neurologist and Clinical Researcher in the Neuromuscular Diseases Unit at Hospital Universitario Virgen del Rocio and Instituto de Biomedicina de Sevilla (HUVR-IBiS), Universidad de Sevilla where she trained as a neurologist. She is one of the curators of the Spanish neuromuscular diseases registry.

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Marie-Christine Ouillade Marie-Christine Ouillade

Board of directors - Association Française contre les Myopathies - AFM
Patient Association Representative

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.

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Miriam  Rodrigues Miriam Rodrigues

New Zealand NMD Registry Curator
Curator of the New Zealand NMD Registry

Miriam Rodrigues is a genetic counsellor whose dedication to rare neuromuscular disorders began when she was appointed Membership Services manager at the Muscular Dystrophy Association of New Zealand (MDA NZ) in 2006.  Launched in 2011 the NZ NMD Registry is an MDA NZ initiative covering all neuromuscular disorders and is co-ordinated by Miriam.

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Rosario dos Santos Rosario dos Santos

Portugese DMD Registry - National Insitute of Public Health
Curator of Portuguese DMD Registry

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Pascale Saugier-Veber Pascale Saugier-Veber

French SMA Registry Curator - University Hospital of Rouen
Curator of French SMA Registry

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Inge Schwersenz Inge Schwersenz

Patient Association - Initiative - Forschung und Therapie fuer SMA Deutsche Gesellschaft fuer Muskelkranke (DGM)
Representative of the German DMD and SMA registries and a patient representative

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Thomas Sejersen Thomas Sejersen

Associate Professor, Astrid Lindgrens Barnsjukhus - Karolinska Institute

Thomas Sejersen (MD, PhD): Head of clinical and research team for neuromuscular disorders in childhood. He is involved in ENMC workshops on DMD and desmin-related myopathies.

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Eduardo Tizzano Eduardo Tizzano

Spanish SMA Registry - Hospital Sant Pau
Coordinator of Spanish SMA registry

Born in La Plata Argentina, Eduardo is a specialist in Pediatrics and Medical Genetics also he is the Principal Investigator of the U-705 CIBERER. He is devoted to diagnosis and research in SMA at the Hospital of Sant Pau of Barcelona, a reference Center for SMA diagnosis from all over Spain.

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Isabela Tudorache Isabela Tudorache

Coordinator of DMD Patient Registry in Romania - Parent Project Association
Coordinator of Romanian DMD Registry

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Sylvie Tuffery-Giraud Sylvie Tuffery-Giraud

Research Engineer - INSERM
Position in Global Database Oversight Committee - Curator of French DMD Registry

Sylvie Tuffery-Giraud (PhD) has 16 years experience in molecular analysis of the dystrophin gene and is particularly involved in mutations leading to splicing defects.

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Jen Wang Jen Wang

China DMD Care and Support Association
Chinese DMD Registry Representative

Jen Wang founded the China DMD Care and Support Association together with other parents of young men with DMD on July 10th 2011. The Association supports families in China and helps them to understand the scientific research and achievements of a therapy for Duchene and Becker Muscular Dystrophy.

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Simon Woods Simon Woods

Bioethicist - Newcastle University
Expertise in ethics and governance

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

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W. Ludo van der Pol W. Ludo van der Pol

Curator of Dutch SMA Registry - University Medical Centre Utrecht
Curator of Dutch SMA Registry

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Jan Verschuuren Jan Verschuuren

Head of Neuromuscular Section, Department of Neurology - Leiden University Medical Center
Position in Global Database Oversight Committee - Curator of Dutch DMD/BMD Registry

Dr Jan Verschuuren is a neurologist at the Department of Neurology of the LUMC, where he heads the neuromuscular section.

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Petr Vondráček Petr Vondráček

Associate Professor of Neurology - University Hospital Brno
Curator of the Czech National DMD/BMD Registry

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Andrea Klein Andrea Klein

Co-founder and responsible for the Swiss registry for SMA and DMD - University Childrens Hospital


 

Moisés  Alatorre Moisés Alatorre

President, Mexico SMA registry curator


Moisés Alatorre is an MD and PhD  born in Guadalajara, Mexico. He is the chair-man and founder of the Asociación Mexicana de Atrofia Muscular Espinal (AMAME) since 2010. AMAME provides free molecular diagnose for SMA for any patient that needs it in México being the national reference center and also develop research about the situation of SMA in his country.

 

Rebecca Leary Rebecca Leary

TREAT-NMD Programme Coordinator

Becca Leary is a member of the TREAT-NMD Secritariat and responsible for the Global SMA and DMD registries.


 
 
12 Apr 2017