The report also provides a detailed analysis of the current Niemann-Pick disease type C marketed drugs and late-stage pipeline drugs. BROOKLYN, BROOKLYN, UNITED STATES, June 25, 2025 /EINPresswire.com ...

In NPC, the disease stems from changes to either the NPC1 or NPC2 genes, which affect the transport of cholesterol and other fats within a cell. Consequently, ...

Niemann-Pick disease type C (NPC) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking ...

New website launched in honor of Rare Disease Day, Feb. 28, 2025 Awareness campaign includes educational resources and testing information for individuals with suspected NPC. CELEBRATION, Fla ...

Scientists report on new insights into the mechanisms of 'Niemann-Pick type C' (NPC), a rare neurodegenerative disease associated with dementia that can manifest as early as childhood. Their ...

In the U.S., only about 900 people are living with NPC. The disease often robs patients of their ability to speak, think clearly, swallow, walk, and move. Many are children.

Findings showed that among patients who also received miglustat (76% of the arimoclomol group and 81% of the placebo group), treatment with arimoclomol slowed NPC disease progression, compared ...

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the necessary transport of ...

NPC is a rare, inherited, progressive and fatal neurodegenerative disease. Jessica Martin pushed for her son to be part of a clinical trial for a new drug called Miplyffa.

In NPC, fats build up in cells because the body can’t remove them. But there is a new drug called Miplyffa that helps protect those cells and slows the disease. “The symptoms stay more ...