ASSOCIATION MONÉGASQUE CONTRE LES MYOPATHIES


MISSIONS

Created in April 2001, Association Monégasque contre les Myopathies - AMM (Monaco Association against Duchenne Muscular Dystrophy) aims at:

  • Collecting funds to finance research, organize scientific roundtables and improve the quality of life of the children, teenagers, and young adults affected by Duchenne Muscular Dystrophy (DMD)
  • Organizing scientific roundtables and International Collaborative Efforts (ICE)
  • Helping people suffering from Duchenne Muscular Dystrophy to benefit from better medical care
  • Increasing public awareness of this genetic disease to better integrate in society these patients
  • Promoting and accelerating the scientific and medical research to find treatments as fast as possible in order to slow down and eventually heal Duchenne Muscular Dystrophy
The international clinicians and researchers working on Duchenne Muscular Dystrophy attending the ICE round table (Monaco, Nov. 27, 2010) with the presence of HSH Prince Albert II
DUCHENNE MUSCULAR DYSTROPHY (DMD)

Duchenne Muscular Dystrophy is a rare genetic illness that affects boys: one birth in 3500.

Progressively serious, it is characterized by a weakening of muscles and leads to lung and cardiac complications that become fatal with the onset of adulthood.

The life of these children is therefore one of increasing dependence on constant care and associated treatments.

Although research has progressed significantly over the last ten years, notably with the tremendous gains in genetic therapy, and thanks to the efforts of charity organizations, there remains a lot to do in order to find a cure for these children.

INTERNATIONAL COLLABORATIVE EFFORT (ICE)
FOR DUCHENNE MUSCULAR DYSTROPHY (DMD) – KEY DATAS

Since Only Watch 2009, € 2.5 million have been distributed by Association Monégasque contre les Myopathies

1 Goal
Overcome enduring hurdles in order to initiate clinical trials using exon skipping strategies

2 Promoters

Association Monégasque contre les Myopathies (A.M.M.)
Duchenne Parent Project France


5 Countries
France, Italy, Switzerland, United Kingdom, U.S.A

15 Institutions
INSERM
University of Milan
University College London
King’s College London
Royal Holloway University London
University of Rome
University of Oxford
University of Bern
University of Pennsylvania
National Institute of Health Bethesda
Institut de Myologie Paris
Imperial College London
Genethon
GénoSafe
INSERM-Transfer

 

18 Partners
Thomas Voit, Myology Institute, Pitié-Salpêtrière Hospital, Paris
Jamel Chelly, Cochin Institute, Cochin Hospital, Paris
Luis Garcia, Pierre & Marie Curie University, Myology Institute, Paris
Gill Butler-Browne, Pierre & Marie Curie University, Myology Institute, Paris
Olivier Danos, Necker Hospital, Paris, & University College, London
Francesco Muntoni & Jennifer Morgan, Institute of Child Health & Great Hormon Street Hospital, London
Kay Davies, Oxford University
George Dickson, Royal Halloway University, London
Dominic Wells, Imperial College, London
Simon Hughes, King’s College, London
Irene Bozzoni, Sapienza University of Rome
Christian Leumann, University of Bern
Yvan Torrente & Giulio Cossu, University of Milan, Maggiore Policlinico Hospital, Milan
Lee Sweeney, University of Pennsylvania, Philadelphia
Robert Kotin, NHBL, National Institutes of Health, Bestheda
Fédor Svinartchouk, Genethon, Evry, France
Muriel Audit, GenoSafe, Evry, France
Anton Ottavi, Inserm-Transfert, Lyon, France

90 Researchers and Clinicians

7 Work packages
1 Evaluation of the therapeutic value of truncated dystrophins completed by exon skipping
2 Design and upgrading optimized-snRNAs for dystrophin rescue
3 Autologous stem cell therapy using LV vectors encoding opt-snRNAs
4 Widespread delivery of AAV vectors encoding opt-snRNAs: Clinical relevance in GRMD
5 Tricyclo-DNA, a promising new chemistry for synthetic antisense oligonucleotides
6 Design of the future clinical trials
7 Management

3 Scientific advisors
Jean-Claude Kaplan (France)
Terry Partridge (UK)
Adrian Trhasher (UK)

1 Scientific coordinator

Luis Garcia (France)