Successful Outcome of Pregnancy in Niemann–Pick Disease Type B: A
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Last updated 17 novembro 2024
A new test for diagnosing Niemann–Pick disease
Successful implementation of classical ketogenic dietary therapy in a patient with Niemann-Pick disease type C - ScienceDirect
Genetic disease and Niemann-Pick disorders: novel treatments and drug delivery systems - ScienceDirect
Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Endpoint Considerations to FacilitateDrug Development for Niemann-Pick Type C (NPC)
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
Niemann–Pick disease - Wikipedia
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
JCM, Free Full-Text
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