Synaptix Bio urgently needs your support to renew the Priority Review Voucher (PRV) program, a vital tool that provides financial incentives to accelerate clinical trials and speed up the FDA review process here in the U.S.

The PRV program was expected to be extended by Congress last December but remains unapproved due to other legislative priorities. Your voice can help change that.

https://everylifefoundation.org/rare-advocates/take-action/

It's hard to believe we are now in our tenth year of advocating for those living with H-ABC/TUBB4A! We have made meaningful headway but the process is slow and complicated. Nevertheless, we continue to drive forward. Here is what we have been doing.

Link to Ten Year Anniversary

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