Events

“Together against CMT”

1^st European Charcot-Marie-Tooth Specialists Conference

9-10 June 2023, at Holiday Inn “Paris Bastille”, PARIS

Editorial

The European Charcot-Marie-Tooth Federation (ECMTF), uniting all of the CMT advocacy groups across Europe, proposes an initiative whose ambition is to bring together all active parties to unite their efforts for the purpose of initiating and stimulating international & interdisciplinary cooperation in research on CMT.

The objective of this Conference is to set up the foundations for a better future of CMT patients and their relatives :

• Develop structured cooperative research to develop effective therapies
• Inform and advise in order to avoid unnecessary late or incorrect diagnosis
• Articulate and stimulate discussions on socio-economic issues of CMT patients 

This novel approach in Europe is intended to bring together researchers, clinicians and other health professionals, with the support of patient advocacy groups, pharmaceutical labs and public partners, as key stakeholders to build a community of specialists.

The three cornerstones of CMT advanced research…

…gathered together with three key stakeholders :

to join their efforts and creativity to find a cure on CMT

Program – Friday 9^th June

• Welcome :
  – Daniel TANESSE, President of ECMTF (CMT-EUROPE)
• Greetings :

• Introduction : « CMT as a common challenge for scientists, clinicians and other health professionals – disciplinary cooperation and potential of interdisciplinary synergies »

1. Bio- and medical sciences : from data (management and analysis) to therapies

Given the potentials of digitization and bio-informatics in basic and applied sciences in the field of genetically caused neuropathies, including CMT, the aim of the workshop is to establish the state of the art developments in this field, and to assess the role that health data generally, as well as genetical data in particular, play for researchers working on the development of causal therapies of CMT.

• Co-moderation : Michael SEREDA & Filippo GENOVESE
• Rapporteur : Alexander ROSSOR
• Speakers : (1) Davide PAREYSON / (2) Francesc PALAU MARTINEZ / (3) Wolfgang Maximilian PERNICE / Helena F. PERNICE

2.  Clinical diagnosis and treatment : Potential of genetic analysis and engineering

The aim of the workshop is to shed some light on the state of the art applied sciences for the diagnosis and treatment of CMT. Clinicians with specific research interests in genetic analysis and engineering are invited to report recent achievements, expectations and perspectives of the progress of science on CMT and its complex genetic causes.

• Co-moderation : Peter DE JONGHE & Arabela ACALINEI
• Rapporteur : Maike DOHRN
• Speakers : (1) Valérie DELAGUE / (2) Angelo SCHENONE / (3) Jan SENDEREK

3.  Therapeutic practice and feedback : Challenges and specific tasks for health professionals

The aim of this workshop is to understand the fundamental role clinical and independent health professionals – physio-, logo-, ergotherapists and nutritionists – play in the treatment as well as in research on CMT, in order to establish how practitioners on the spot contribute not only to the well-being of each patient but also to the study of CMT through their daily observations and collected data on the effect of specific therapies (or new drugs?).

• Co-moderation : Carsten SCHRÖTER & Alexandre HOYAU
• Rapporteur : Roberto CANNATARO
• Speakers : (1) Cale FABIEN / (2) Gita RAMDHARRY / (3) Joshua BURNS

• Podium : Highlights from the working groups (10’ each Rapporteur)
• Comments: Pavel Seeman (t.b.c.)
• General discussion : key insights and learnings / open questions

Venue : Maison de l’Amérique Latine – Paris, 7^th Arrondissement

Venue : Maison de l’Amérique Latine – Paris, 7^th Arrondissement

Program – Saturday 10th June

1. Cooperation : Establishing a “European CMT Research Association (ECRA)”

With a view to institutionalizing closer cooperation among research centers and scientists in Europe we would hope that the workshop can come up with some guidelines and provisions for how scientists and researchers, clinicians and other health professionals with a scientific aspiration can organize synergies of their efforts, eventually by setting up a European CMT Research Association aiming at stimulating the process of CMT research.

• Co-moderation : Laurent MAGY & Dany MAES
• Rapporteur : Liliane MASSADE
• Presenters : (1) Gian Maria FABRIZI / (2) Teresa SEVILLA MANTECÓN / (3) Lisa REINECKE

2. Data management for CMT : Towards an Integrated European CMT Patient Registry

Research on CMT often suffers from the very limited access to data; as CMT is a rare disease even for specialized clinicians CMT patients are rare. This means researchers depend on data from many other centers specialized on CMT in order to reach a minimum sample of data to base their studies upon. Patient registries, either general ones on neuromuscular diseases or specialized for CMT, exist in some countries. But they are organized according to different criteria and standards, so there is no interoperability and their usefulness is limited.

The aim of the workshop is to determine the need for quality data to be made available to researchers as a basis for promising research efforts, and to develop proposals for how to have a coherent system of compatible registries which makes available all the data needed.

• Co-moderation : Alessandra BOLINO & Marco VAN DER LINDEN
• Rapporteur : Andrea CORTESE
• Presenters : (1) Jean-Christophe ANTOINE / (2) Simone THIELE / (3) Teresinha EVANGELISTA

3. Mobilizing the Patients : Scientists, Clinicians and other health professionals expectations and advice

CMT patients often seem to be reluctant to take a stake in the development of research and treatment of their disease, let alone raising public awareness and understanding in politics for their special problems and needs. The aim of this workshop is to find methods and mechanisms for mobilizing the patients in their own interest. Mobilization means that we would wish for more of them to join our patient advocacy organizations and efforts of self-help. It also means closer cooperation with the scientists, clinicians and other health professionals in studies and by making their data available to researchers, e.g. through patient registries or apps. There may be many other ideas on how to use the potential that the mobilization of patients can offer   for the benefit of more efficient research and, finally of a better quality of life for the patients themselves.

• Co-moderation : Tanya STOJKOVIC & Inbal BEN DAVID
• Rapporteur : Signe VAETH
• Presenters : (1) Raluca Loana TELEANU / (2) Kleopas A. Kleopa / (3) Nicolas TRICAUD

• Podium : Reports from the working groups (10’ each Rapporteur)
• Comments : An American Perspective : Katherine FORSEY (CRO of CMTA)
• Final plenary discussion

Contacts