Niemann-Pick disease causes a variety of medical problems, and it often advances rapidly. The symptoms and effects of all of the variants of Niemann-Pick disease result from the buildup of ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... the cells, and causes a range of symptoms that ...

Xenpozyme (olipudase alfa) the first and only approved therapy for ASMD, a group of genetic disorders also known as Niemann-Pick disease ... on patients' lives and causes early death: as young ...