Successful Outcome of Pregnancy in Niemann–Pick Disease Type B: A

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PDF) Successful Outcome of Pregnancy in Niemann–Pick Disease Type B: A Case Report and Review of Literature

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Genetic disease and Niemann-Pick disorders: novel treatments and drug delivery systems - ScienceDirect

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II

Consensus clinical management guidelines for acid sphingomyelinase deficiency (Niemann–Pick disease types A, B and A/B), Orphanet Journal of Rare Diseases

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

PDF] Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician

PDF) Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: A 5-year update from the UK clinical database