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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Niemann-Pick disease


Other Names for this Disease
  • Sphingomyelin lipidosis
  • Sphingomyelinase deficiency
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Treatment


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How might Niemann-Pick disease type A be treated?

There is no specific treatment for this disease. Supportive care from the following specialists may be helpful for managing the symptoms:[1][2] 

  • A pulmonologist for respiratory problems
  • A cardiologist for heart problems
  • Liver and spleen specialists
  • Nutritionists
  • Physical therapists
  • A gastroenterologist
  • Learning specialists

You can learn more about ongoing research efforts to better understand the natural history of this condition and identify treatment options in the Research section of our web page.



Last updated: 4/17/2014

References
  1. Niemann Pick Disease. National Organization for Rare Disorders (NORD). 2006; http://www.rarediseases.org/rare-disease-information/rare-diseases/byID/93/viewAbstract. Accessed 4/17/2014.
  2. Treatment Options for NPA. National Niemann-Pick Disease Foundation, Inc.. http://www.nnpdf.org/npdisease_07.html. Accessed 4/17/2014.


Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Niemann-Pick disease. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • The Research Portfolio Online Reporting Tool (RePORT) provides access to reports, data, and analyses of research activities at the National Institutes of Health (NIH), including information on NIH expenditures and the results of NIH-supported research. Although these projects may not conduct studies on humans, you may want to contact the investigators to learn more. To search for studies, click on the link and enter the disease name in the "Terms Search" box. Then click "Submit Query".