A global network of non-profit organisations supporting those affected by Niemann-Pick diseases

Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies...

AQNEURSA® (levacetylleucine) recommended for EU approval by the CHMP to treat Niemann-Pick Disease Type C

Zevra Submits Arimoclomol MAA to EMA for Niemann-Pick Disease Type C Treatment