Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety 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 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 ...

Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposiumEight abstracts discussing data and clinical ...

In 2017, neurologists at New York Presbyterian/ Columbia University’s movement clinic diagnosed Daniel with Niemann-Pick disease Type C (NPC): a lysosomal storage disorder caused by NPC1 mutations.

A number of first-in-disease, first-in-class and first-in-modality drugs approved last year have redefined therapeutic areas, ...

The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick disease type C, a rare genetic disease, in adults and children weighing at least 15 kg ...