Understanding the real-life experiences of individuals and families affected by Niemann-Pick Disease Type C (NPC) is essential to improving care, research, and future treatment development. A new research initiative led by Janney Wang from the University of Sydney is aiming to do exactly that—by listening directly to the voices of people living with NPC, their carers, and their families. This work focuses on capturing the everyday realities of living with NPC, including: The biggest...

Melbourne, Australia – April 23, 2026 The Australian NPC Disease Foundation is proud to announce the publication of Australia’s first national clinical guidelines for Niemann-Pick disease type C (NPC). This milestone marks an important step forward in improving the recognition, diagnosis and care of individuals living with NPC across Australia. A collaborative national effort In 2024, ANPDF funded the NPC Care and Guidelines Project, bringing together more than 40 clinicians, allied health...

For many families in the rare disease community, social media has become a powerful advocacy tool. It helps raise awareness, connect with other families, influence research, and push for change. For years, parents and caregivers have been the voices telling these stories — often long before their children were able to understand them. But what happens when your affected child grows up and starts using social media themselves? This moment can bring pride, uncertainty, and sometimes discomfort....