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 US regulator has cleared Miplyffa (arimoclomol ...

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients. IntraBio's ...

More information: Lina Dinkel et al, Myeloid cell–specific loss of NPC1 in mice recapitulates microgliosis and neurodegeneration in patients with Niemann-Pick type C disease, Science ...

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 ...

It’s a characteristic sign of a rare genetic disease called Niemann Pick disease type C. “Gelastic” refers to laughter, and “cataplexy” is a sudden, temporary episode of muscle weakness ...

An announcement from Zevra Therapeutics ( (ZVRA)) is now available. Zevra Therapeutics has announced the commercial availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for ...

In 2024, multiple drugs received complete response letters (CRLs), sometimes unrelated to the safety and efficacy of the drug ...

“We will do anything to raise awareness of any rare disease,” Harry said. Niemann-Pick Disease is a rare illness the Koujaians are very familiar with. Harry’s daughter and Alex’s sister ...

Make smarter investments with weekly expert stock picks from the Smart Investor ... developments in the treatment landscape for Niemann-Pick disease type C (NPC) with the recent approval of ...

In the journal “Science Translational Medicine”, scientists from DZNE and LMU Hospital report on new insights into the mechanisms of “Niemann-Pick type C” (NPC), a rare neurodegenerative ...

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 ...