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.

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Drug Repurposing

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.

👉 Learn more about the Drug Repurposing Study

Family Webinar on Initial Drug Repurposing Findings

The Foundation to Fight H-ABC partnered with Unravel Biosciences to conduct a computational systems biology study using patient-derived molecular data from individuals with TUBB4A-related leukodystrophy. This work was designed to explore the molecular pathways underlying the disease and identifying potential therapeutic opportunities.  These findings were recently presented in a webinar to the patient community.  See attached link to the webinar, and more details with FAQ.

.👉 An update

Unravel Findings & FAQ

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.

👉 Learn more about Rare X and how to contribute

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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.

👉 Find out how to enroll in the Natural History Study

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Speech and Language in individuals with H-ABC:

The Translational Centre for Speech Disorders is running a project looking at speech and language outcomes in individuals aged 6 months to adulthood with H-ABC.

👉 Find out how to enroll in this international study

Need Help Getting Started?
We’re here to walk you through any step of the process.

Email us: contact@h-abc.org