Scientists report on new insights into the mechanisms of 'Niemann-Pick type C' (NPC ... that can permanently halt the disease. "Although we know the genetic causes of NPC, the mechanisms relevant ...

Niemann-Pick disease affects multiple systems throughout the body. It has many symptoms, and the severity varies from case to case. Rather than being one disease, Niemann-Pick has four distinct types.

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 ...

"Although we know the genetic causes of NPC ... microgliosis and neurodegeneration in patients with Niemann-Pick type C disease, Science Translational Medicine (2024). DOI: 10.1126 ...

Piracha, MDLipoid pneumonia (LP) is a rare, non-infectious condition that occurs when oil or fat (lipids) molecules enter the ...

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 ...

In the journal "Science Translational Medicine", scientists from DZNE and LMU Hospital report on new insights into the mechanisms of "Niemann-Pick ... disease. "Although we know the genetic causes ...

Zevra Therapeutics has announced the commercial availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick Disease Type C (NPC). This breakthrough offers a new ...

“Although we know the genetic causes of NPC ... microgliosis and neurodegeneration in patients with Niemann-Pick type C disease ...