As 2024 draws to a close, we take a moment to reflect on the incredible work accomplished by the Australian NPC Disease Foundation (ANPDF) and the unwavering support we have received from our community. It has been a year of growth, collaboration, and significant milestones, all aimed at improving the lives of those impacted by Niemann-Pick Disease Type C (NPC).
A Year of Advocacy and Progress
This year, ANPDF continued its advocacy efforts for the approval of NPC treatments in Australia. We’ve made significant strides, with a particular focus on AQNEURSA (levacetylleucine) and Miplyffa (arimoclomol). We also connected with several up-and-coming research organisations to continue exploring potential therapy options for our families. Thanks to the tireless work of our board, staff, and supporters, we’ve raised awareness within the Australian medical community and the general public.
We also hosted our Advocating for Treatments in Australia Webinar, which provided an important platform for patients, families, and medical professionals to discuss the current treatment landscape with Rare Voices Australia, and the ongoing efforts to gain approval for these essential therapies in Australia.
Successful Fundraising Initiatives
Alongside an incredible year of generous donations, our fundraising initiatives have seen great success this year. Highlights include:
The Silver Raffle, which raised an incredible $2,130, supporting research and patient care.
The Inaugural Poker Tournament at the Benalla Bowls Club, raising $2,300.
Ellie's special Kando Martial Arts Fundraiser, in which she raised $1,600 with her family and co-hosted a very special self-defence class.
The Minds in Motion Charity Cocktail Fundraiser, held at The Timberyard in June 2024, which brought together our supporters for a vibrant evening of cocktails, conversation, and philanthropy, raising over $53,000 for NPC research and patient support.
These contributions have gone directly toward funding research, the Guidelines project and supporting families affected by NPC, and bringing us closer to our ultimate goal: accessible treatments for all.
Community Engagement Initiatives
Our community continues to be at the heart of everything we do. This year, we launched our Care and Guidelines Project, a comprehensive guide for managing NPC. The project is on track to publish its first National Care and Guidelines document in June 2025, a crucial step forward in ensuring that patients receive the best care possible.
In addition, we hosted our Annual Niemann-Pick Conference, which brought together medical professionals, researchers, and families for valuable discussions on the latest advancements in Niemann-Pick research and care. The conference is a key event in advancing awareness and collaboration within the NPD community, and we are proud of the progress made this year.
Looking Ahead: A Bright Future for 2025
As we look toward 2025, we’re excited about the continued progress in Niemann-Pick awareness, treatment access, and research. We will be welcoming new and returning committee members following the upcoming Annual General Meeting in February 2025, and we look forward to working with them to further our mission.
Thank you for being part of this journey with us. Your support makes all the difference, and we are incredibly grateful for your contributions.
How You Can Help in 2025
As we enter a new year, there are many ways you can continue to support our cause:
Together, we can create a brighter future for all those impacted by Niemann-Pick Disease in Australia and around the world.