Rechercher des projets européens

The goal of BATCure is to advance the development of new therapeutic options for a group of rare lysosomal diseases - neuronal ceroid lipofuscinoses (NCL) or Batten disease. There are > thousand affected across Europe, with a combined incidence of c.1:100 000. The NCLs are devastating and debilitating genetic disorders that mainly affect children, who suffer progressive dementia and motor decline, ...

...dical interventions for a disadvantaged and very low frequency group in Europe – children and young people with Juvenil Neuronal Ceroid Lipofuscinosis (JNCL), also called Spielmeyer-Vogt disease or Batten disease. The disease is characterized by a developmental course including blindness, epilepsy, speech problems, cognitive regression/dementia, motor coordination problems and emotional reactions ...