Niemann-Pick Disease, Type C: Diagnosis, Management and Disease-Targeted Therapies in Development
Elizabeth Berry‐Kravis
Topics & Concepts
MedicineNiemann–Pick disease, type CDiseaseDystoniaDysphagiaCognitive declineAtaxiaExome sequencingNeurodegenerationPediatricsDysarthriaBioinformaticsDementiaPathologyPsychiatrySurgeryGeneticsBiologyGeneMutationLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusChild Nutrition and Feeding Issues