Research

Researchers at Mayo Clinic investigate drugs in the treatment for Niemann-Pick disease type C and study the natural history of this disease through an international database, as well as a longitudinal study of cognition in Niemann-Pick disease type C.

Publications

See a list of publications from Mayo doctors on Niemann-Pick disease on PubMed, a service of the National Library of Medicine.

Research Profiles

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Feb. 16, 2017
References
  1. Patterson MC. Overview of Niemann-Pick disease. http://www.uptodate.com/home. Accessed Oct. 31, 2016.
  2. Patterson MC, et al. Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: An observational cohort study. Orphanet Journal of Rare Diseases. 2015;10:65. http://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0284-z. Accessed Oct. 31, 2016.
  3. Niemann-Pick disease. Merck Manual Professional Version. https://www.merckmanuals.com/professional/pediatrics/inheriteddisordersofmetabolism/Niemannpickdisease. Accessed Oct. 31, 2016.