About
Spinal muscular atrophy caused by impairment of the SMN (survival of motor neurons) gene induces the death of neurons that control the activity of skeletal muscles. Currently about 1500 patients live in France with this disease. In its most severe form, it causes the death of most children before the age of 2 years. Revolutionary treatments recently developed by Roche, Novartis and Biogen have increased the life expectancy of children but have created a new clinical form of the disease which is crucial to characterize in order to allow good management of treated patients and consider future clinical trials to address functional deficits that persist despite treatment. Muscular atrophy is a major component of these deficiencies.
The RHU SMART (SMA Muscle Atrophy Remediative Therapy), led by Pr Laurent Schaeffer and endowed with funding of 9.4 million euros, has two main objectives:
– Describe the pathology of treated patients and identify relevant biomarkers through a multicenter and international natural history study.
– Develop a process for screening molecules to allow the emergence of treatments for muscle atrophy. SMART brings together 6 laboratory teams (B. Chazaud, J. Courchet, V. Gache, V. Jacquemond, R. Mounier, L. Schaeffer), the teams of D. Freyssenet and G. Millet from the LIBM in Saint-Etienne and that of F. Charbonnier from the University of Paris, 2 local biotechs (Cytoo, EnyoPharma) and 2 large industrial groups (Roche and Sysnav).
Our ambition for the SMART project is to initiate the creation of a clinical investigation platform for neuromuscular diseases that would combine academic research, therapeutic trials and access to innovative molecules in the field of neuromuscular diseases.
This network has been labeled by the Ministry of Health (Investissement d’avenir) and INSERM to bring neurology closer to immunology and develop innovative approaches for inflammatory diseases of the nervous and neuromuscular system
The kick off meeting of the RHU SMART took place on 27 May 2022, at the Rockefeller Faculty of Medicine and Pharmacy, Lyon.
40 participants in person and by videoconference (including the ANR) attended the kick-off meeting.
Different presentations were given in the morning. After the buffet, the participants met in different workshops: natural history, fatigue, screening and medicinal chemistry.
More information about RHU SMART is available on its website.
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