In July, NBC News featured a powerful story on H-ABC/TUBB4A leukodystrophy, highlighting the challenges faced by affected families and the urgent need for research. This national coverage brings much-needed awareness and shines a light on the hope and resilience within the community.

The 2025 ULF Annual Family Conference brought together families, clinicians, and researchers from across the leukodystrophy community. The Foundation to Fight H-ABC hosted a special session on H-ABC/TUBB4A, featuring leading experts, research updates, advocacy voices, and powerful family testimonials. This gathering offered knowledge, connection, and hope for everyone navigating the H-ABC journey.

[Read more & watch the full symposium →]

Watch the N-Lorem podcast and learn about the first child's journey finding and receiving a treatment for the Tubb4a gene mutation using an ASO developed by N-Lorem.

👉 Link to n-lorem

Join us June 27-28 at the ULF Family Conference in Chicago, and learn more about what we are doing at the mini symposium dedicated to H-abc/Tubb4a

On February 27-29, 2025, we joined a very productive three day conference at the Children's Hospital of Philadelphia. Key advocacy groups, scientists, researchers and biotechs came together from across the globe, to discuss various leukodystrophies.

Here we are featured with Dan Williams of Synaptix Bio and Dr. Adeline Vanderver who is the lead clinician at CHOP tied to H-abc/Tubb4a.

See link to the event, the H-abc/Tubb4a session starts right around hour 6.

https://m.youtube.com/watch?v=whPJJkBujK4&t=21218s&pp=2AHipQGQAgE%3D

A day, actually an entire week dedicated to raising awareness around rare disease on the Hill. We joined approximately 1000 advocates from across 50 states who descended upon Congress and the Senate making our case for the following:

• Bringing public and congressional awareness to the unique needs of rare diseases

• Addressing the challenges due to the few affected within each disease and delays in getting diagnosis

• Extending the Priority Review Voucher (PRV) and Accelerated Access Care Act

• Addressing the lack of specialty care, the fact patients have to travel outside of state, many of whom depend on Medicaid which won't allow coverage across state lines

• Delays in treatment and loss of life as a result

Camden is an 18 year old fun loving and energetic teenager who celebrates Christmas all year round and the magic, hope, and joy that comes with the season. He enjoys time with his family, Florida State football, touching trucks, listening to contemporary Christian music, and is somehow the President of his school's Good Choice Club.

LINK TO CAMDEN’S STORY

We are so excited to share that our fundraiser in February was an amazing hit! We raised approximately $50,000!! What an amazing accomplishment and we could not do it without the amazing talent of our fundraising team Bob and Mickie Ziarko, their family and friends, the amazing H-abc families who joined us on stage, and The Village people! Thank you everyone for your incredible support!

See LINK to the news media coverage!