Registries Oversight Committee
About the 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 committee is chaired by the TREAT-NMD activity leader.
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 01-01-2006) and valued at more than 2,000 Euro.
Anna Ambrosini - Fondazione Telethon, Italy
Personal Information of OC member
Name: Anna Ambrosini
Affiliation/Employer: Fondazione Telethon, Italy.
Role in relation to global registries: Vicepresident of the Association of the Italian registry of patients with neuromuscular diseases-onlus and representative on behalf of the association by the TGDOC.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Sarah Baumeister - German DMD and SMA Registry
Personal Information of OC member
Name: Sarah Baumeister
Affiliation/Employer: Friedrich-Baur-Institute, University of Munich
Role in relation to global registries: German National Registry
Disclosure Statement
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Christophe Béroud - Global Patient Registry Organiser
Personal Information of OC member
Name: Christophe Béroud
Affiliation/Employer: University of Montpellier, Montpellier University Hospital, INSERM U827, FR
Role in relation to global registries: TREAT-NMD work package leader for patient registries
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Fabrizia Bignami - EURODIS Representative
Personal Information of OC member
Name: Fabrizia BIGNAMI
Affiliation/Employer: EURORDIS
Role in relation to global registries: In charge of EURORDIS advocacy activities regarding patients’ registries for rare diseases
Disclosure Statement
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Ria Broekgaarden - Dutch Patient Organisation Representative
Personal Information of OC member
Name: Ria Broekgaarden
Affiliation/Employer: VSN
Role in relation to global registries: IPA is co owner of a global survey/registry of data.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Filippo Buccella - Italian DMD Registry Curator
Personal Information of OC member:
Name: Filippo Buccella
Affiliation/Employer: Parent Project onlus
Role in relation to global registries: Coordinator
I and/or my spouse/partner have no financial interest with any organization that
Nick Catlin - UK DMD Registry Representative
Personal Information of OC member
Name: Nick Catlin
Affiliation/Employer: Action Duchenne
Role in relation to global registries: Registry Manager
Disclosure Statement
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Kevin Flanigan - US DMD Registry Representative
Personal Information of OC member
Name: Kevin Flanigan
Affiliation/Employer: University of Utah
Role in relation to global registries: We have established a registry of DMD Patients
Disclosure Statement
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Pat Furlong - Patient Organisation Representation
Personal Information of OC member
Name: Pat Furlong
Affiliation/Employer: Parent Project Muscular Dystrophy (PPMD).
Role in relation to global registries: Provide financial support for DuchenneConnect.org
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Lauren Hache - CINRG Database
Personal Information of OC member
Name: Lauren Hache
Affiliation/Employer: CINRG / University of Pittsburgh
Role in relation to global registries: Curator for CINRG Database
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Dr Ian Max Huxham - SMA Europe Representative
Personal Information of OC member
Name: Dr Ian Max Huxham
Affiliation/Employer: SMA Europe
Role in relation to global registries: Patient Organisation Representative
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Serap İnal - Turkish DMD and SMA Registry Curator (Istanbul)
Personal Information of OC member
Name: Serap İnal
Affiliation/Employer: Prof.Dr. PT; Istanbul University, Turkey.
Role in relation to global registries: Patient Registry in Turkey.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Jacqueline Jackson - USA SMA Registry
Personal Information of OC member
Name: Jacqueline M. Jackson
Affiliation/Employer: Indiana School of Medicine
Role in relation to global registries: Oversight Committee, International Spinal Muscular Atrophy Patient Registry, USA.
Disclosure Statement
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Dr Pierre-Yves Jeannet - Swiss SMA and DMD Registry
Personal Information of OC member
Name: Dr Pierre-Yves Jeannet
Affiliation/Employer: Centre Medical Universitaire Vaudois, Lausanne, Switzerland
Role in relation to global registries: Swiss National Registry
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Anna Kaminska - Polish DMA and SMA Registry
Personal Information of OC member
Name: Anna Kaminska
Affiliation/Employer: Medical University of Warsaw, Department of Neurology
Role in relation to global registries: Coordinator
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
A. Ayse Karaduman - Turkish DMD and SMA Registry Curator (Ankara)
Personal Information of OC member
Name: A. Ayse Karaduman
Affiliation/Employer: Hacettepe University
Role in relation to global registries: DMD and SMA registry curator in Ankara Turkey
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Veronica Karcagi - Hungarian DMD and SMA Registry
Personal Information of OC member
Name: Dr. Veronica Karcagi
Affiliation/Employer: Biologist, Human Geneticist, PhD; National Institute of Environmental Health (NIEH), Hungary
Role in relation to global registries: Database Curator
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Dr Janbernd Kirschner - TREAT-NMD Clinical Trials Coordination Centre
Personal Information of OC member
Name: Dr. Janbernd Kirschner
Affiliation/Employer:
Klinik II: Neuropädiatrie und Muskelerkrankungen
Zentrum für Kinder- und Jugendmedizin
Universitätsklinikum Freiburg
Mathildenstraße 1
D - 79106 Freiburg
Jaana Lähdetie - Finnish DMD and SMA Registry
Personal Information of OC member
Name: Jaana Lähdetie
Affiliation/Employer: Turku University Central Hospital
Role in relation to global registries: Head of Finnish TREAT-NMD Patient Registry
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Hanns Lochmuller - TGDOC Chair
Personal Information of OC member
Name: Hanns Lochmuller
Affiliation/Employer: University of Newcastle, UK
Role in relation to global registries: TREAT-NMD activity leader for patient registries; chair of oversight committee
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Vitaliy Matyushenko - Ukrainian SMA Registry
Personal Information of OC member
Name: Vitaliy Matyushenko
Affiliation/Employer: President of Charitable Foundation “Children with SMA”, Ukraine
Role in relation to global registries: Curator national DB
Violeta Mihaylova - Bulgarian DMD and SMA Registry
Personal Information of OC Member
Name: Violeta Mihaylova
Affiliation/Employer: University Hospital “Alexandrovska”, Department of Neurology, Sofia
Role in relation to global registries: Bulgarian TREAT-NMD Registries
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Vanessa Rangel Miller - DuchenneConnect DMD registry in USA
Personal Information of OC member
Name: Vanessa Rangel Miller
Affiliation/Employer: DuchenneConnect / Emory University
Role in relation to global registries: Coordinator and genetic counsellor for DuchenneConnect.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Ian Murphy - Spanish DMD Registry
Personal Information of OC member
Name: Ian Murphy
Affiliation/Employer: Self Employed
Role in relation to global registries: Organiser
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Dr Harumasa Nakamura's - Japanese DMD Registry
Personal Information of OC member
Name: Harumasa Nakamura
Affiliation/Employer: National Center of Neurology and Psychiatry (NCNP)
Role in relation to global registries: Responsible for DMD patient registry in Japan.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Marie Christine Ouillade - Patient Association Representative
Personal Information of OC member
Name: Marie Cjristine Ouillade
Affiliation/Employer: AFM, France
Role in relation to global registries: Patient Association
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Pascale Saugier-Veber - French SMA Registry Curator
Personal Information of OC member
Name: Pascale Saugier-Veber
Affiliation/Employer: University Hospital of Rouen and Faculty of Medicine of Rouen, France
Role in relation to global registries: Molecular curator of the SMA Database.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
M. Rosário dos Santos - Portugese DMD Registry
Personal Information of OC member
Name: M. Rosário dos Santos
Affiliation/Employer: Centro de Genética Médica J. Magalhàes, I.N.S.A
Role in relation to global registries: Curator of National Registry
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Thomas Sejersen - Swedish DMD and SMA Registry
Personal Information of OC member
Name: Thomas Sejersen
Affiliation/Employer: Karolinska Institute / Karolinska University Hospital
Role in relation to global registries: Oversight Committee Member
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Eduardo Tizzano - Spanish SMA Registry
Personal Information of OC member
Name: Eduardo Tizzano
Affiliation/Employer: Department of Genetics, Hospital Sant Pau, Barcelona, Spain
Role in relation to global registries: Coordinator of SMA registry in Spain.
Slyvie Tuffery-Giraud - French DMD Registry Curator
Personal Information of OC member
Name: Sylvie Tuffery-Giraud
Affiliation/Employer: Montpelier 1 University, INSERM U827, France.
Role in relation to global registries: DMD Registry Curator
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Peter Van den Bergh - Belgian DMD Registry
Personal Information of OC member
Name: Peter Van den Bergh
Affiliation/Employer: Université catholique de Louvain, Brussels, Belgium.
Role in relation to global registries: Coordinator of the Belgian Neuromuscular Disease registry and the Belgian TNMD DMD database.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Jan Verschuuren - Dutch DMD/BMD Registry
Personal Information of OC member
Name: Jan Verschuuren
Affiliation/Employer: Leiden University Medical Center (LUMC)
Role in relation to global registries: Curator of the Dutch DMD/BMD registry.
Disclosure Statement:
I and/or my spouse/partner have a financial interest/arrangement with one or more organization(s), including but not limited to the organization(s) supporting this activity, that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Further details:
Self or Spouse? Self
Name of Organization: Prosensa
Nature of relevant financial relationship: Trial support in 2007
What was received? Financial support for execution of the antisense trial with PRO-051 was given to the LUMC.
For what role? Principal Investigator
Petr Vondracek - Czech DMD registry
Personal Information of OC member
Name: Petr Vondracek
Affiliation/Employer: Department of Paediatric Neurology, University Hospital Brno, Czech Republic.
Role in relation to global registries: Curator of the national DMD/BMD registry.
Disclosure Statement :
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
Simon Woods - Chair of Project Ethics Council
Personal Information of OC member
Name: Simon Woods
Affiliation/Employer: Newcastle University UK
Role in relation to global registries: Oversight Committee member: offering expertise in ethics and governance.
Disclosure Statement:
I and/or my spouse/partner have no financial interest with any organization that could be perceived as a real or apparent conflict of interest in the context of the subject of this activity.
