THE DUCHENNE MUSCULAR DYSTROPHY
The life of these children is therefore one of increasing dependence on constant care
and associated treatments.
The Duchenne Muscular Dystrophy is a rare genetic illness that affects boys: one birth in 3,500.
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 MONACO ASSOCIATION AGAINST DUCHENNE MUSCULAR DYSTROPHIES
Created in April 2001, Association Monégasque contre les Myopathies aims at
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 (DMD).
The Monaco-based Charity's missions:
- Collecting funds to finance research,
- Organising and financing the scientific research program
namedInternational Collaborative Effort for DMD (ICE for DMD),
- Helping people suffering from DMD to benefit from better
- Improving the quality of life of the children, teenagers
and young adults affected by DMD,
- Increasing public awareness of this genetic disease to
better integrate in society these patients.
SYNTHENA Biotech (Bern, Switzerland)
In July 2012, AMM and a small group of Scientists created Synthena, a Swiss biotech
for managing the development of a new class of oligonucleotides for exon skipping, the tricyclo-DNA in order to make these compounds available for clinical trial.
Centre Scientifique de Monaco (CSM)
AMM is also developing a partnership with the Centre Scientifique de Monaco (CSM),
which has decided recently to adjoin a new research laboratory on muscle dystrophies and handicap.
Université de Versailles Saint-Quentin (UVSQ)
AMM finances the salaries Luis Garcia team : 2 research scientists, 2 engineers, 1
In the near future, AMM’s aim is to foster close ties between the CSM in Monaco,
Synthena in Bern and research teams at the University of Versailles saint-Quentin working on therapeutic approaches for DMD, to combine their skills and knowledge.