Highlights from the 4th National Symposium on Bone Marrow Failure Syndromes

From 23–25 May 2025, Maddie’s Vision was honoured to host the 4th National Symposium on Bone Marrow Failure Syndromes held at the Victorian Comprehensive Cancer Centre (VCCC). This important event brought together Australia’s leading clinicians, scientists, researchers, allied health professionals, industry partners, patients and families—along with international experts—for three days of knowledge sharing and collaboration focused on Bone Marrow Failure Syndromes.

The Symposium opened with a powerful Opening Plenary, featuring presentations from Dr Marcin Wlodarski (St. Jude Children’s Research Hospital, Memphis), patient advocate Ross Forsyth, and Anna McMahon, a parent sharing her family’s journey. This session also celebrated Maddie Riewoldt’s Vision’s 10 Years of Impact, with presentations from CEO Amy Coote and Tele-Support Service Nurse Mei Ling Yeh, followed by a documentary highlighting a decade of impact in research and support.

Across Saturday and Sunday, attendees heard from three international guest speakers, Dr Marcin Wlodarski and Dr Marta Derecka (St. Jude Children’s Research Hospital, Memphis), and Dr Paula Río (Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Madrid), as well as 24 national presenters, 13 of whom were from the Maddie’s Vision Centre of Research Excellence (CRE). Topics ranged from basic science, translational and clinical research, gene therapy, allied health research, and patient case studies.

The event hosted 135 attendees with strong engagement across all days. Thanks to the outstanding support of our sponsors and partners, we were able to offer complimentary registration to all participants.

Organised by a dedicated Steering Committee chaired by Dr Lucy Fox and supported by the Maddie’s Vision Executive Team, the symposium secured sponsorship from valued partners including Novartis, Royal Melbourne Hospital Foundation, Takeda, Sobi, and Jazz Pharmaceuticals. Additional in-kind support from Zouki catering and other event partners helped us deliver an exceptional event experience.

Feedback from attendees was overwhelmingly positive:

• 100% of survey respondents were satisfied with the balance of scientific, clinical, and case study content.
• The event received an overall rating of 9.4/10, with content rated 9/10 for relevance and engagement.
• 87% of attendees broadened their professional networks, and 75% reported identifying new collaboration or innovation opportunities.

“I truly enjoyed the mix of basic and translational research. I greatly appreciated the presentations from health care providers who interact with Bone Marrow Failure Syndrome patients. The bone marrow niche session was one of the best I attended in recent months.”

“Thank you for providing a professional and high-quality setting for meaningful professional learning.”

“I liked the balance of patient stories, clinical work and research. It also felt very inclusive, and very positive. Everyone’s passion to improve lives was very clear.”

“One of the most well-organised events I’ve attended. Kudos to the MRV team!”

The success of the 2025 Symposium is a testament to the strength of the Bone Marrow Failure Syndrome community and the collective commitment to improving outcomes for patients and families. We extend our sincere thanks to all our speakers, sponsors, attendees, and organisers who helped make this year’s symposium such a memorable and impactful event.

Together, we continue to build momentum in research, care, and connection for today and the future.

We would like to acknowledge and extend our sincere gratitude to the following sponsors for their support and contribution towards this event: