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 ...

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 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 ...