Your Involvment Isn’t Just Important
It’s Essential
How Families Can Help Accelerate H-ABC Research
Your Voice, Your Story, Your Data – They All Matter
Families affected by H-ABC/TUBB4A are central to driving research forward. Your participation in key studies and data platforms helps scientists understand the condition better, speed up drug development, and move us closer to a cure. Whether you’re newly diagnosed or further along in your journey, you can play a direct role in shaping the future of treatment.
Below are three powerful ways to get involved — each backed by researchers, specialists, and patient advocacy leaders.
Drug Repurposing Study
Help identify existing medications that might help children with H-ABC.
We’re working with top researchers to test already-approved drugs for their potential to treat symptoms of H-ABC/TUBB4A. Your input and participation can help researchers identify candidates faster and reduce the time to treatment.
Rare X Data Collection
Share your medical journey to help researchers worldwide.
By securely sharing your child’s data through Rare X, you contribute to a growing global database that accelerates scientific discovery. This platform ensures researchers have access to the most up-to-date, real-world information on H-ABC.
Natural History Study
Help researchers track how H-ABC progresses over time.
Understanding how the condition unfolds is key to building better treatments. The Natural History Study collects clinical information from families like yours to inform future trials, therapies, and care recommendations.
Need Help Getting Started?
We’re here to walk you through any step of the process.
Email us: contact@h-abc.org