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Results 1 - 10 of 13 for "Niemann-Pick" "disease," type A
  1. Levacetylleucine 
    ... at least 33 pounds (15 kg) who have Niemann-Pick disease type C (NPC; an inherited disease in which a ... to treat the neurologic symptoms in patients with Niemann-Pick disease type C.
    https://medlineplus.gov/druginfo/meds/a624063.html - Drugs and Supplements
  2. Arimoclomol 
    ... 2 years of age and older who have Niemann-Pick disease type C (NPC; an inherited disease in which a ... works to treat neurologic symptoms in patients with Niemann-Pick disease type C.
    https://medlineplus.gov/druginfo/meds/a624065.html - Drugs and Supplements
  3. NPC2 gene 
    ... the NPC2 gene have been found to cause Niemann-Pick disease type C2. This type of Niemann-Pick disease is ... feeding problems. The NPC2 gene mutations that cause Niemann-Pick disease type C2 reduce or eliminate NPC2 protein activity, which ...
    https://medlineplus.gov/genetics/gene/npc2 - Genetics
  4. NPC1 gene 
    ... the NPC1 gene have been found to cause Niemann-Pick disease type C1. This type of Niemann-Pick disease is ... causing the tissue and organ damage seen in Niemann-Pick disease type C1. More About This Health Condition Niemann-Pick ...
    https://medlineplus.gov/genetics/gene/npc1 - Genetics
  5. Niemann-Pick disease 
    ... signs and symptoms of the condition.Infants with Niemann-Pick disease type A usually develop an enlarged liver and spleen ( ... mental abilities and movement (psychomotor regression). Children with Niemann-Pick disease type A also develop widespread lung damage (interstitial lung ...
    https://medlineplus.gov/genetics/condition/niemann-pick-disease - Genetics
  6. SMPD1 gene 
    ... the SMPD1 gene have been found to cause Niemann-Pick disease types A and B. These types of Niemann-Pick ... complete loss of enzyme function tend to cause Niemann-Pick disease type A. In the Ashkenazi (eastern and central European) ...
    https://medlineplus.gov/genetics/gene/smpd1 - Genetics
  7. Lipid Storage Diseases From the National Institutes of Health (National Institute of Neurological Disorders and Stroke)  
    Leukodystrophies/Specifics ... Leukodystrophies ... Genetic Brain Disorders/Specifics ... Genetic Brain Disorders ... Tay-Sachs Disease/Related Issues ... Tay-Sachs Disease ... Lipid ...
    https://www.ninds.nih.gov/.../disorders/lipid-storage-diseases - External Health Links
  8. Niemann-Pick disease 
    ... Organization for Rare Disorders -- rarediseases.org/rare-diseases/niemann-pick-disease-type-c ... provider if you have a family history of Niemann-Pick disease and you ... Developmental problems Feeding problems Poor weight gain
    https://medlineplus.gov/ency/article/001207.htm - Medical Encyclopedia
  9. Volunteer Stories From the National Institutes of Health (National Institutes of Health)  
    Clinical Trials/Specifics ... Clinical Trials ... National Institutes of Health ... From the National Institutes of Health ... Comments from volunteers participating in clinical ...
    https://www.nih.gov/.../personal-stories - External Health Links
  10. Lipid Metabolism Disorders (National Library of Medicine)  
    Metabolism is the process your body uses to make energy from the food you eat. Food is made up of proteins, carbohydrates, and fats. Chemicals in your digestive ...
    https://medlineplus.gov/lipidmetabolismdisorders.html - Health Topics
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