Cyclo Therapeutics has shared topline results from its Phase 3 TransportNPC study, which included Australian participants. While the study’s main endpoint was not statistically significant, important findings—including a reported survival benefit for children with infantile-onset NPC—warrant close attention and further review.

Xenpozyme® (olipudase alfa) has been listed on the Life Saving Drugs Program (LSDP) for patients with Acid Sphingomyelinase Deficiency (ASMD).

The blood-brain barrier is a roadblock in NPC treatment, but new strategies aim to safely deliver therapies directly to the brain.

FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.

New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!