PATHOLOGIES

Amyotrophic Lateral Sclerosis

ALS, also referred as Lou Gerhig disease in the US or maladie de Charcot in France, is the most common degenerative disease of the motor neuron system. It affects about 150 000 people worldwide. ALS results from the systematic dismantling of the motor neuron system required for voluntary movement in multiple regions of the body. Early symptoms of ALS include stiff muscles, muscle twitches, and gradual increasing weakness and muscle wasting. Limb-onset ALS begins with weakness in the arms or legs, while bulbar-onset ALS begins with difficulty speaking or swallowing. The cause of ALS is unknown, although a family history of the disease is seen in about 10% of patients. ALS-associated genetic factors include C9ORF72, TARDBP (TDP-43), SOD1 and FUS genes, which make up to 70% of all familial ALS cases. There is no cure for ALS today. The drugs riluzole (Rilutek®) and edavarone (Radicava®), approved in some regions, provide limited efficacy. Therefore, new agents halting or slowing down the disease process are desperately needed.