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Results 1 - 8 of 8 for "Niemann-Pick" "disease," type C1
  1. NPC1 gene 
    ... the NPC1 gene have been found to cause Niemann-Pick disease type C1. This type of Niemann-Pick disease is characterized ... causing the tissue and organ damage seen in Niemann-Pick disease type C1. More About This Health Condition Niemann-Pick disease, ...
    https://medlineplus.gov/genetics/gene/npc1 - Genetics
  2. Niemann-Pick disease 
    ... survive into adulthood.The signs and symptoms of Niemann-Pick disease types C1 and C2 are very similar; these types differ only in their genetic cause. Niemann-Pick disease types C1 and C2 usually become apparent in childhood, although ...
    https://medlineplus.gov/genetics/condition/niemann-pick-disease - Genetics
  3. Levacetylleucine 
    ... at least 33 pounds (15 kg) who have Niemann-Pick disease type C (NPC; an inherited disease in which a certain ... to treat the neurologic symptoms in patients with Niemann-Pick disease type C.
    https://medlineplus.gov/druginfo/meds/a624063.html - Drugs and Supplements
  4. Arimoclomol 
    ... 2 years of age and older who have Niemann-Pick disease type C (NPC; an inherited disease in which a certain ... works to treat neurologic symptoms in patients with Niemann-Pick disease type C.
    https://medlineplus.gov/druginfo/meds/a624065.html - Drugs and Supplements
  5. 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
  6. 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
  7. Niemann-Pick disease 
    ... Organization for Rare Disorders -- rarediseases.org/rare-diseases/niemann-pick-disease-type-c/
    https://medlineplus.gov/ency/article/001207.htm - Medical Encyclopedia
  8. NPC2 gene 
    ... Hendriksz CJ, Sedel F, Walterfang M, Kolb SA. Niemann-Pick disease type C symptomatology: an expert-based clinical description. Orphanet J ...
    https://medlineplus.gov/genetics/gene/npc2 - Genetics