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An Overview of Niemann-Pick Disease
Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety of ...
pharmaphorum4mon
FDA clears first drug for Niemann-Pick disease
Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease. The US regulator has cleared Miplyffa (arimoclomol ...
ALZFORUM26d
Microglia Drive Neurodegeneration in Niemann-Pick Type C
In 2017, neurologists at New York Presbyterian/ Columbia University’s movement clinic diagnosed Daniel with Niemann-Pick disease Type C (NPC): a lysosomal storage disorder caused by NPC1 mutations.
Calgary Herald9d
Nieman: Lessons learned from families over four decades
Dr. Nieman is the founder of Centre 70 Pediatrics. He has written monthly columns for the Herald since 1999.
pharmaphorum2y
Xenpozyme is first FDA-approved drug for rare disease ASMD
Xenpozyme (olipudase alfa) the first and only approved therapy for ASMD, a group of genetic disorders also known as Niemann-Pick disease, in which a fatty substance called sphingomyelin builds up ...