Niemann-Pick disease type A

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:

  • 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 on 05-01-20

Name: Canadian Society for Mucopolysaccharide and Related Diseases Inc. (Canadian MPS Society) #218-2055 Commercial Drive Vancouver, BC V5N OC7
Phone: 604-924-5130 Toll Free: 800-667-1846 Fax : 604-924-5131 Email: Url:
Name: National Tay-Sachs and Allied Diseases Association 2001 Beacon Street Suite 204
Brighton, MA, 02135, United States
Phone: 617-277-4463 Toll Free: 800-90-NTSAD (906-8723) Fax : 617-277-0134 Email: Url:
Name: Metabolic Support UK 5 Hilliards Court Sandpiper Way
Chester Business Park
Chester, CH4 9QP, United Kingdom
Phone: 0845 241 2173 Toll Free: 0800 652 3181 Email: Url:
Name: Hide and Seek Foundation for Lysosomal Storage Disease Research 6475 East Pacific Coast Highway Suite 466
Long Beach, CA, 90803 , United States
Toll Free: (844) 762-7672 Email: Url:
Name: National Niemann-Pick Disease Foundation, Inc. P.O. Box 49 401 Madison Avenue, Suite B
Ft. Atkinson, WI, 53538, United States
Phone: 920-563-0930 Toll Free: 877-CURE-NPC (287-3672) Fax : 920-563-0931 Email: Url:
Niemann-Pick disease Genetics Home Reference (GHR). January 2008; Reference Link NINDS Niemann-Pick Disease Information Page National Institute of Neurological Disorders and Stroke (NINDS). October 6, 2011; Reference Link Niemann Pick Disease National Organization for Rare Disorders (NORD). 2006; Reference Link Treatment Options for NPA National Niemann-Pick Disease Foundation, Inc.. Reference Link

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The RareGuru disease database is regularly updated using data generously provided by GARD, the United States Genetic and Rare Disease Information Center.

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