Treatment Options for Niemann-Pick Type C

Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, describes dif

Niemann-Pick disease treatment: a systematic review of clinical trials.

FDA says Hold Off on Zavesca (Miglustat) approval. « Niemann-Pick Children's Fund, Inc

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders - Papandreou - 2022 - Developmental Medicine & Child Neurology - Wiley Online Library

Parents of twins with Niemann-Pick find a treatment

Recommendations for the detection and diagnosis of Niemann-Pick disease type C

Niemann-Pick disease type C as a neurovisceral disease. Schematic

IJMS, Free Full-Text

Summer 2022 Newsletter – NNPDF

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Frontiers Lysosomal and Mitochondrial Liaisons in Niemann-Pick Disease

Recommendations on the diagnosis and management of Niemann-Pick disease type C - ScienceDirect

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library