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May 16, 2022

CONFERENCE Innovative, combinatory therapies for neuromuscular diseases


Free and open to everyone
Mandatory registration
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CONFERENCE Innovative, combinatory therapies for neuromuscular diseases CONFERENCE Innovative, combinatory therapies for neuromuscular diseases

Genopole and the LBEPS are pleased to invite you to a conference on therapeutic innovations for neuromuscular diseases and the perspectives of combinatory therapies.

We will be pleased to welcome researchers from the San Raffaele Scientific Institute in Milan, the Mondor Institute for Biomedical Research and Inserm teams, as well as researchers from the Genethon, I-Stem and LBEPS laboratories on our campus.

When:
May 16, 2022  @ 10:00 – 16:00

Where:
Salle de conférence of GIP Genopole
20, rue Henri Desbruères
91000 Evry-Courcouronnes

>>> Access Map

Abstract:

Neuromuscular diseases encompass a wide variety of rare diseases with different etiologies, but all of them affect either motor neurons, neuromuscular junctions or muscles. Moreover, they cause disabling motor deficits and may even lead to death. The development of new therapeutic strategies for such diseases is a scientific and technological challenge.

The conference aims to explore this issue, not from a mono-pathological point of view, but rather an intersecting one, considering the different diseases of the motor unit. The furtherance of knowledge on these heterogeneous pathologies will open new perspectives on treatment.
Therapeutic innovations are based on pharmacological, cellular, gene, and physical therapies, that, when crossed or combined, may provide promising paths forward.

The conference will energize a discussion on the benefits and limitations of developing combinatory therapies for diseases affecting neuromuscular function

Program:

10:00 – Introduction

10:10 – Session 1. Myopathies

The alarmin HMGB1 links oxidative stress and inflammation in muscular dystrophies: a novel therapeutic avenue
Emilie Vénéreau (Tissue Regeneration and Homeostasis Unit, San Raffaele Scientific Institute, Milan)

Co-administration of Simvastatin does not potentiate the benefit of gene therapy in the mdx mouse model for Duchenne Muscular Dystrophy
Dr. David Israeli (Progressive muscular dystrophies Team, Généthon)

Molecular therapies in inflammatory diseases
Pr. François-Jérôme Authier (Centre expert de pathologie neuro-musculaire, CHU Henri Mondor, IMRB, Biologie du système neuromusculaire, Créteil)

Cell therapy for muscular dystrophies: reasons for lack of efficacy and possible solutions.
Pr. Giulio Cossu (Cell therapy for myopathies Unit, San Raffaele Scientific Institute, Milan)

11:40 – Session 2. Neuropathies

Stem-cell derived cellular models of Charcot Marie Tooth disease: therapeutic perspectives?
Dr. Nicolas Tricaud (Equipe Maladies des fibres myélinisées, I-Stem)

The development of targeted therapies for Charcot Marie Tooth 1A disease
Dr. Liliane Massade (Thérapies ciblées pour les neuropathies périphériques, U1195 INSERM Maladies et hormones du système nerveux)

Oxysterols/Liver X Receptor: a journey exploring their roles in myelination and the treatment of peripheral neuropathies
Pr. Charbel Massaad (Myelination and Nervous system pathologies, UMR-S1124, INSERM, Université Paris Cité)

12:50 – Lunch

14:00 : Session 3. Motor neuron diseases

Human pluripotent stem cells for neuromuscular diseases
Cécile Martinat (UMR-S 861, I-Stem)

Combinatory therapeutic innovation for spinal muscular atrophy
Olivier Biondi (Precision exercises for dystrophies and their therapies, LBEPS, Université d’Evry Paris-Saclay, Université Paris Cité)

15:00 : Round Table

– Pharmacological, gene, cellular, and physical therapies: how can innovations improve the efficacy of treatments for neuromuscular diseases?
– Is it possible to foresee the use of identical therapies for different neuromuscular disorders?
– Should combinatory approaches be developed?
– What benefits can be expected?

With the contribution of the speakers and Pr David ADAMS, Head of the Neurology Department of the CHU Bicêtre (APHP), coordinator of Nnerf.

Steering Committee:

  • Roxane Brachet, Academic Partnerships Manager, Genopole
  • Olivier Biondi, Associate Professor, LBEPS, University of Evry Paris-Saclay, University of Paris Cité
  • Véronique Le Boulc’h, Science and Citizen Communications Manager, Genopole

This event is free and open to everyone.
Mandatory registration

Registration Form

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With the support from
Région île de France