
A rare diagnosis—hope starts here.
Changing the future for children with H-ABC
What is H-ABC?
H-ABC (Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum) is a rare and progressive neurological disorder caused by mutations in the TUBB4A gene. It typically begins in infancy or early childhood and affects the brain's ability to form the protective myelin sheath around nerve cells.
Over time, children with H-ABC may lose the ability to sit, stand, walk, speak, or swallow independently. This condition lies at the most severe end of the TUBB4A-related leukodystrophy spectrum.
There is no cure—yet. But through research, advocacy, and community support, we are working to change that future.
The Foundation to Fight H-ABC/TUBB4A was established in 2015 with a clear and urgent mission:
To raise awareness, support families affected by H-ABC/ TUBB4A-related leukodystrophy, and drive research toward effective treatments—and ultimately, a cure—for Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC) and TUBB4A-related leukodystrophy.
Fuel Research and Hope
Your donation directly supports groundbreaking research in gene therapy and other promising treatments for H-ABC and TUBB4A-related leukodystrophies. Every dollar moves us closer to a world with better outcomes—and eventually, a cure.
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Be Part of the Movement
Join our events, share our story, or volunteer to raise awareness in your community.
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Every Share Makes a Difference
H-ABC is rare—but the more people who know about it, the greater our collective impact. Share our stories, follow us on social media, or invite others to learn about this condition and our mission.
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A Cure is Within Reach—We Need Your Help to Get There
By supporting the Foundation to Fight H-ABC/TUBB4A, you help:
Fund groundbreaking research in gene therapy and other promising treatments
Connect families to critical resources, specialists, and support networks
Raise global awareness of this devastating and rare condition
Let’s give every child with H-ABC and other TUBB4A-related leukodystrophies a fighting chance.