Niemann-Pick disease is a rare genetic disorder that renders the body incapable of metabolizing cholesterol and other lipids inside of cells. The result is a buildup of these fatty substances in ...

Arimoclomol is a small molecule designed to amplify and sustain the cellular production of heat shock proteins, particularly HSP70, which targets protein misfolding and improves lysosomal function.

A total of 92 gene variants were sequenced, 41 of which were novel. A new report offers novel insights into links between the clinical, biochemical, and genetic characteristics 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 medical ...

Regulators upheld their earlier decision after reexamination, saying the substance does not meet the criteria for new active ...

Denise and Rickey Miller are pleading for help, knowing time could be running out for their 21-month-old son Woodrow, who is diagnosed with a rare disease called Niemann-Pick Disease Type-C. Denise ...

Despite the wide variety of presentations in Niemann-Pick type C (NP-C), there is a common element—early cognitive decline. However, little is known about the cognitive impairment in patients with ...

CHESTERFIELD, Va. (CNN Newsource/WTVR/WKRC) - A Disney song helps take 8-year-old Abby Alvey to another world, a world where she's free of the stress and issues related to her terminal diagnosis of ...

A Valley 2-year-old who is fighting a rare and fatal disease is now showing improvement with an experimental drug. Woodrow Miller suffers from Niemann-Pick Type C disease, described as a form of ...

A boy who was expected to live for less than a year because of a rare genetic condition has died at the age of three after contracting COVID. Sebastian Moroney was born with a severe form of ...

DUBLIN--(BUSINESS WIRE)--The "Global Niemann-Pick Disease Market and Competitive Landscape - 2020" report has been added to ResearchAndMarkets.com's offering. This report provides comprehensive ...