Niemann-Pick type B is caused by a defect in the SMPD1 gene, which results in reduced production of Acid Sphingomyelinase, which causes sphingomyelin to build up in the cells, which in turn ...

A new study in the journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... other lipids within cells. That leads to the ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who ...

It’s been just six months since Zevra Therapeutics scored an FDA nod for Miplyffa as the first approved treatment for the rare lysosomal storage disorder Niemann-Pick disease type C (NPC), but ...

a group of genetic disorders also known as Niemann-Pick disease, in which a fatty substance called sphingomyelin builds up in cells and caused them to die, disrupting tissue and organ function.

A new study in the journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease ... on better recognition of cancer cells by the body's own immune system.

The Chargers made Niemann a sixth-round pick in 2021, and he spent his first four seasons with the team. He has played 64 games with three starts, seeing action on 363 defensive snaps and 1,298 on ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick ...