Periodic Reporting for period 1 - Treat-HSP (Development of new therapy for rare motor neuron diseases)
Período documentado: 2018-11-01 hasta 2020-04-30
The ERC-POC funding allowed us to demonstrate that a method decreasing the expression of a key enzyme for the synthesis of gangliosides, GM3 synthase, slowed down the progression of symptoms and slightly decreased neuronal death in an animal model of the disease. This proof of concept allowed us to team up with a biotech to pursue this project and translate it into treatment for patients in a few years.