EVENT

“Together against CMT”

1^st European Charcot-Marie-Tooth Specialists Conference

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

Editorial

“Together against CMT” is the watchword of our initiative to bring together European scientists, clinical researchers, and therapists specialized in CMT, to join their efforts in developing effective causal therapies for CMT.

The European CMT Federation uniting most of the CMT advocacy groups across Europe feels particularly called to stimulate and promote interdisciplinary cooperative research in the interest of all patients affected.

We simultaneously strive to mobilize the patients to cooperate more closely with the specialists involved and to contribute as they can (in studies and with their data) to broaden the basis for successful research.

The different groups of specialists no doubt each already have their own gatherings and schemes of cooperation, but for the first time we call on you to create synergies and innovative ideas in a joint conference.

And we are happy to include representatives of the profession and of organizations from overseas so as to learn from their experience and stimulate cooperation also on the global level.

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

CONFERENCE REPORT

CMT Summit in Paris by Ingolf Pernice, Berlin – To read the conference report, click here

CONFERENCE VIDEOS

The entire conference has been recorded, and you can follow the plenaries, the workshops and
the concluding session if you click on the videos each title at the place indicated (hour: minute:
second)

Opening Plenary

Welcome:

00.15 Daniel Tanesse, President of CMT-EUROPE (ECMTF)
06:50 Ingolf Pernice, Vice-President of CMT-EUROPE, Conference Manager
Video message: 16:05 Stella Kyriakides, EU Commissioner for Health and Food Safety
Greetings: 21:50 Alexandre Méjat, Director AFMTelethon & EURORDIS
27:23 Agnès Firmin Le Bodo, ministre déléguée chargée des Professions de santé

Introductions: CMT as a common challenge for scientists, clinicians and other health professionals – disciplinary cooperation and potentials of interdisciplinary synergies.
36:00 European projects/perspectives : Shahram Attarian, Marseilles
51:23 American projects/perspectives : Stephan Züchner, Miami

Working Groups on Substance: Three disciplinary approaches to problem solving

1. Bio- and medical sciences: From data (management and analysis) to therapies
   1:03:39 co-moderation: Michael Sereda / Filippo Genovese

• Presenters: (1:06:00) Davide Pareyson (1:29:30) Francesc Palau (1:54:56) Wolfgang
  M. Pernice/Helena F. Pernice
• Rapporteur: Rafael Sivera (2:16:00)

Working Groups on Substance: Three disciplinary approaches to problem solving

2. Clinical diagnosis and treatment: Potentials of genetic analysis and engineering
   00:48 co-moderation : Peter De Jonghe / (40:20) Arabela Acalinei

• Presenters : (03:00) Valérie Delague; (43:08) Angelo Schenone
• Rapporteur : Maike Dohrn

Working Groups on Substance: Three disciplinary approaches to problem solving

3. Therapeutic practice and feedback: Challenges and specific tasks for therapists
   co-moderation : Carsten Schröter / Alexandre Hoyau

• Presenters : (00:16) Fabian Cale (15:06) Gita Ramdharry (34:14) Mel Mandarakas
• Rapporteur : Roberto Cannataro (01:11:30)

Plenary II Substance: Three disciplinary approaches to problem solving (chair: Mary Reilly, London)

• Podium: Highlights from the working groups
  Presenters : (02:00) Rafael Sivera (02:00), Maike Dohrn (13:20), Roberto Cannataro (23:12)
• Comments: Pavel Seeman
• (40:10) General discussion: key insights and learnings – and the open questions

Working Groups on Strategies: Creating synergies of three approaches

1. Cooperation: Establishing a « European CMT Research Association (ECRA)»
   co-moderation : (00:15) Laurent Magy / (01:16) Simon Bull

• Presenters : (02:16) Teresa Sevilla Mantecón (12:40) Lisa Reinecke (30:46) Tim VanGansewinkel
• 45:50 : Discussion
• Rapporteur : Andrea Gangfuss

Working Groups on Strategies: Creating synergies of three approaches

2. Data management for CMT: Towards an Integrated European CMT Patient Registry
   co-moderation : (01:36) Alessandra Bolino / (00:15) Marco van der Linden

• Presenters : (03 :20) Simone Thiele (29:30) Teresinha Evangelista (46:44) Jean Christophe Antoine
• 56:20 Discussion
• Rapporteur : Andrea Cortese

Working Groups on Strategies: Creating synergies of three approaches

3. Mobilizing the Patients: Scientists’, Clinicians’ and Therapists’ expectations and advice
   co-moderation : Tanya Stojkovic / Inbal Ben David

• Presenters : (00:15) Oana-Aurelia Vladacenco (21:40) Kleopas A. Kleopa (59:50) Nicolas Tricaud
• Rapporteur : Signe Vaeth

Plenary III Strategies: Creating synergies of three approaches (president: Michaela Auer-Grumbach)

• Podium: Reports from the working groups
  • Presenters : (00:55) Andrea Gangfuss, (08:54) Andrea Cortese, (23:00) Signe Vaeth
• Comments: An American Perspective, Katherine Forsey
• Final plenary discussion

General Conclusions and Perspectives (1:13:50)

• Vincent Timmerman: Take-aways of the Conference
• Filippo Genovese: Award ceremony for the winner of the poster contest
• Ingolf Pernice: Acknowledgements, perspectives and goodbye

INTERVIEWS POST-EVENT

The interviews present the different perspectives discussed at the conference and with the participants, having the experts interviewed by patient representatives.

Interview 1

Poster Contest Winners : Jihane Gadacha, Doctoral student & Nathalie Bernard-Marissal, Doctor

Interviewed by Stephan Züchner, Professor for Human Genetics and Neurology & Alexandre Méjat, International Affairs Officer, Scientific Department – AFM Téléthon

Interview 2

Specialist : Vincent Timmerman, Scientist

Interviewed by Inbal Ben David, Head of Operations, Innovation & Sales at Bayer & Arabela Acalinei, President of Neuro Move CMT Association

Interview 4

Specialist : Maike Dohrn, Neurologyst

Interviewed by Catherine Forsey, Chief Research Officer at CMTA Maria Paola Cadeddu, Business & Integration at Arch Assoc Manager

Interview 5

Specialist : Andrea Cortese, Scientist 

Interviewed by Simon Bull, CEO of CMT UK

Contacts