Search Results 11-20 of 12264 for niemannpick

  1. A Study to Evaluate N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) - Mayo Clinic

    The purpose of this study is to assess the safety and effectiveness of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).

  2. Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C - Mayo Clinic

    Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, genetic disease leading to impaired lysosomal function and neurodegeneration causing ...

  3. Emerging therapies for pediatric lysosomal disorders - Mayo Clinic

    Niemann-Pick type C. Mayo Clinic patients have access to two medications — arimoclomol and miglustat — for the treatment of Niemann-Pick type C (NPC), a ...

  4. ORAL AZ-3102 in Patients With GM2 Gangliosidosis or Niemann Pick Type C Disease - Mayo Clinic

    ORAL AZ-3102 in Patients With GM2 Gangliosidosis or Niemann Pick Type C Disease · Share · Facebook · Twitter.

  5. Inherited metabolic disorders - Symptoms and causes - Mayo Clinic

    Maple syrup urine disease. Metachromatic leukodystrophy. Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS). Niemann-Pick.

  6. Levacetylleucine (oral route) - Side effects & dosage - Mayo Clinic

    Levacetylleucine is used to treat the neurologic symptoms of Niemann-Pick disease type C (NPC). This medicine is available only with your doctor's ...

  7. Arimoclomol (oral route) - Side effects & dosage - Mayo Clinic

    For Niemann Pick disease type C: Adults and children 2 years of age and older—Dose is based on body weight and must be determined by your doctor. Weighing more ...

  8. Early Access Program With Arimoclomol in US Patients With NPC - Mayo Clinic

    About this study. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, ...

  9. Gastroenterology and Hepatology - Conditions treated - Mayo Clinic

    Niemann-Pick disease; Nodular regenerative hyperplasia; Nonalcoholic fatty liver disease; Nonalcoholic steatohepatitis (NASH); Obesity · Pancreatic cancer ...

  10. Enlarged spleen (splenomegaly) - Symptoms and causes - Mayo Clinic

    People who have Gaucher disease, Niemann-Pick disease, and several other inherited metabolic disorders affecting the liver and spleen; People who live in or ...