Cerliponase alfa changes the natural history of children with neuronal ceroid lipofuscinosis type 2: The first French cohort
Bastien Estublier, Aline Cano, Célia Hoebeke, Samia Pichard, Didier Scavarda, Isabelle Desguerre, Stéphane Auvin, B. Chabrol
Topics & Concepts
MedicineEnzyme replacement therapyNatural history studyNatural historyCohortNeuronal ceroid lipofuscinosisPediatricsDiseaseInternal medicineLysosomal Storage Disorders ResearchTrypanosoma species research and implicationsGlycogen Storage Diseases and Myoclonus