GeneDx Holdings WGS announced a sponsored genetic testing program with Zevra Therapeutics to improve diagnosis for patients suspected of having Niemann-Pick Disease Type C (NPC), a rare and ...

Barbara Lazarus’ two sons, David and Daniel, were healthy and active kids, but things changed in their early 20s. David developed severe psychiatric symptoms, which were diagnosed as schizoaffective ...

Six years ago, Austin sisters Belle and Abby Andrews were diagnosed with Niemann-Pick Type C1, a rare genetic disorder sometimes called childhood Alzheimer’s. Belle, who was 6 at the time, was falling ...

A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...

At its July 2025 meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended granting a marketing authorization in the European Union for Aqneursa ...

Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym: ...