Join the sweet side of holiday cheer with these recipes! Some of our NPC Warriors are able to take a treatment to help delay the onset of symptoms caused by Niemann-Pick type C. However, to manage the digestive side affects caused by this medicine, they maintain a diet that restricts their intake of disaccharides (double sugars) including sucrose, lactose, and maltose. Thanks to new sugar-free brands emerging in Australia, low-disaccharide snacks are becoming more readily ava

A seven-episode series following Aussies with rare genetic conditions, as they search for answers, options and community. This year, one of our NPC families participated in the Diagnosis Day series produced by Melbourne Genomics and Genetic Support Network Victoria. It is a fantastic series demonstrating the journey and patient-perspective of various families with different genetic conditions. Meet six Aussie families, including our NPC Warrior Noah, who live with ultra-rare

"IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 - NCT05163288)." Read the full article here: 70+ NPC patients were screened across 13 trial sites in Australia, Europe, United Kingdom and United States. The trial was able to be over-enrolled by approximately 130% and the data-rea