Niemann-Pick DiseaseSymptoms, Doctors, Treatments, Advances & More
Learn About Niemann-Pick Disease
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However, there may be experts who have treated this or similar conditions in our Find a Doctor section and research may be available in our Latest Advances section.
The Children's Hospital At Montefiore
Melissa Wasserstein is a Pediatrics provider practicing medicine in Bronx, New York. Dr. Wasserstein is rated as an Elite provider by MediFind in the treatment of Niemann-Pick Disease. She is also highly rated in 7 other conditions, according to our data. Her clinical expertise encompasses Acid Sphingomyelinase Deficiency (ASMD), Niemann-Pick Disease, Reticulohistiocytoma, and Splenomegaly.
Eugen Mengel practices practicing medicine in Hochheim, Germany. Mr. Mengel is rated as an Elite expert by MediFind in the treatment of Niemann-Pick Disease. He is also highly rated in 19 other conditions, according to our data. His clinical expertise encompasses Niemann-Pick Disease, Acid Sphingomyelinase Deficiency (ASMD), Non-Langerhans-Cell Histiocytosis, and Reticulohistiocytoma.
MN - Neuro Pediatric
Marc Patterson is a Pediatric Neurologist practicing medicine in Rochester, Minnesota. Dr. Patterson is rated as an Elite provider by MediFind in the treatment of Niemann-Pick Disease. He is also highly rated in 20 other conditions, according to our data. His clinical expertise encompasses Niemann-Pick Disease, Polycystic Lipomembranous Osteodysplasia with Sclerosing Leukoencephalopathy, Leukodystrophy, and CACH Syndrome.
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But symptoms can vary among different people with the disease. Some may have seizures, while ot...
Summary: An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis