9 NOVEMBER 2019. 2PM - GENEVA

The aim of the Monegasque Association against Muscular dystrophy (AMM) is to support scientific and medical research on Duchenne muscular dystrophy (DMD), a devastating myopathy affecting one in 3500 male births.

Over the last 15 years, the AMM has organized “Round Tables in Monaco” on the progress of research for DMD, and thanks to the Only Watch charity auction, has sponsored many laboratories worldwide to explore the most promising therapeutic avenues (stem cell therapy, which consists engrafting fresh cells to rebuild skeletal muscles; gene therapy for targeting a replacement gene coding the missing dystrophin into the whole musculature; and finally antisense strategies to rescue the mutated dystrophin gene product throughout exon-skipping).

 

Without going into detail, the AMM funding has allowed forefront research laboratories to recruit young scientists, to acquire cutting-edge equipment and to improve significantly their operational resources. AMM has also initiated SYNTHENA in Switzerland and SQY Therapeutics in France, two companies dedicated in drug discovery and preclinical development at the service of the patient's cause, which are integrated in an international consortium involving academic partners.

 

At present, we are focussing on several complementary axes of research:

 

  • Understanding muscle plasticity and physiopathology of the Duchenne muscular dystrophy
  • Investigating cardiac complications to improve life-span of patients
  • Developing new splice-switching compounds to rescue mutated mRNAs and restore dystrophin in all affected tissues of the body
  • Developing new gene vectors that could be re-administered without recourse to immunosuppression protocols

 

We do our best to keep this alliance up and to progress as soon as possible towards the clinical testing of effective and safe drugs considering that our disease needs a chronic treatment. This goal seems to be nearing completion with a novel generation of synthetic compounds developed by SYNTHENA in cooperation with SQY and several academic entities.

 

As things stand, this novel compound is safe in rodents and non-human primates, and according to mouse models of the disease it allows dystrophin rescue and functional improvement in skeletal muscles, heart and brain following few weeks of treatment. Nonetheless, in the shorter term, we are supporting top quality medical studies on the management of the cardiac complications through the retrospective analysis of enriched databases “genotype/treatment/phenotype” to evaluate the benefit, if there is, of certain combinations of drugs prescribed to patients over the last decade, as well as we contribute on demand to ongoing clinical trials to accelerate the implementation of the best care practices.

 

We are conscious that there is still much to be done and that our initiative needs, more than ever, help and support.

 

Luc Pettavino

 

President - Association Monégasque contre les Myopathies

Founder / Organizer - Only Watch

 

Monaco, March 2019

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