Read the full Community Update from Mandos Health here: Mandos Health has shared an October update highlighting exciting progress in Niemann-Pick type C (NPC) research. Their investigational therapy, adrabetadex , continues to show promise, with new data suggesting a substantial survival benefit  in infantile-onset NPC1 and evidence of improved cholesterol trafficking . Their survival study was recognised as an “Abstract of Distinction”  at the American Neurological Associati

Dr Ya Hui Hung presented at the 2025 INPDA Bi-Annual Meeting. Australia is emerging as a hub of innovation in rare disease research, and...

DL33, developed by Meizon Innovations, is preparing for the next stage of testing — and could one day change the future of childhood dementia. 💜

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