Association Française Niemann Pick

AFNP - MetabERN

Different Niemann-Pick C1 Genotypes Generate Protein Phenotypes that Vary in their Intracellular Processing, Trafficking and Localization

Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

Association Française Niemann Pick

Cyclophosphamide hydrate (Cyclophosphamide monohydrate), Alkylating Agent

Justin Hopkin - Board Member - Uplifting Athletes

RareMD - Rare Disease Organizations

Impaired tyrosine phosphorylation in asmase-/- T lymphocytes after TCR

Niemann–Pick C disease: functional characterization of three NPC2 mutations and clinical and molecular update on patients with NPC2 - Verot - 2007 - Clinical Genetics - Wiley Online Library