Project mission
The REMODEL project focuses on the development of innovative disease models for a broad spectrum of very severe rare neuromuscular diseases without definitive therapeutic options, such as Duchenne muscular dystrophy (DMD) and amyotrophic lateral sclerosis (ALS). The project was designed to provide a global picture of neuromuscular junction (NMJ) alterations that contribute to the pathological signature of rare neuromuscular diseases and to uncover the clinical importance of the balance between mutations common genetic mutations and clinical manifestations and heterogeneity in disease progression of the disease.