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Article

A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B.

Pediatrics - 1 Aug 2008

McGovern Margaret M, Wasserstein Melissa P, Giugliani Roberto, Bembi Bruno, Vanier Marie T, Mengel Eugen, Brodie Scott E, Mendelson David, Skloot Gwen, Desnick Robert J, Kuriyama Noriko, Cox Gerald F

Abstract excerpt

OBJECTIVE: The objective of this study was to characterize the clinical features of patients with Niemann-Pick disease type B and to identify efficacy end points for future clinical trials of enzyme-replacement therapy. METHODS: Fifty-nine patients who had Niemann-Pick disease type B, were at lea...

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Identifiers

PMID: 18625664
DOI: 10.1542/peds.2007-3016
PMCID: PMC2692309

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-18625664-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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