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Why Early-Stage Funding Changes Everything in Rare Disease — Including H-ABC/TUBB4A
Why Early-Stage Funding Changes Everything in Rare Disease — Including H-ABC/TUBB4A
Rare Disease Is Not Small — It Is a System Hiding in Plain Sight
Rare Disease Is Not Small — It Is a System Hiding in Plain Sight
The Bridge
The Bridge
Five Years of Impact: Honoring Bob Ziarko
Five Years of Impact: Honoring Bob Ziarko
Giving Tuesday & the Fight Against H-ABC: Why Your Gift Matters
Giving Tuesday & the Fight Against H-ABC: Why Your Gift Matters
Fall Wins for the H-ABC Community: Progress, People & Momentum
Fall Wins for the H-ABC Community: Progress, People & Momentum
Foundation to Fight H-ABC Welcomes Two Leading Experts to Advisory Team
Foundation to Fight H-ABC Welcomes Two Leading Experts to Advisory Team
Take Action: Support Renewal of the Rare Pediatric Disease Priority Review Voucher (PRV)
Take Action: Support Renewal of the Rare Pediatric Disease Priority Review Voucher (PRV)
Ten Year Anniversary!
Ten Year Anniversary!
NBC News Highlights the Fight Against H-ABC
NBC News Highlights the Fight Against H-ABC
Families Met and Families Shared at the 2025 ULF Conference.
Families Met and Families Shared at the 2025 ULF Conference.
First N-1 Treatment for Tubb4a gene mutation
First N-1 Treatment for Tubb4a gene mutation
May 28. 2025 ~ Upcoming ULF Conference
May 28. 2025 ~ Upcoming ULF Conference
March 12. 2025 ~ Global Leukodystrophy Conference
March 12. 2025 ~ Global Leukodystrophy Conference
March 12. 2025 ~ Let’s talk about the Rare Disease Day 2025
March 12. 2025 ~ Let’s talk about the Rare Disease Day 2025
Meet Camden!
Meet Camden!
What a Groovy Fundraiser!
What a Groovy Fundraiser!
January 13, 2024 ~ Drug Repurposing
January 13, 2024 ~ Drug Repurposing
Wishing Connor well!
Wishing Connor well!
Meet Ben - Living Joyfully with Purpose!
Meet Ben - Living Joyfully with Purpose!
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Featuring another family recently diagnosed........
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Testimony directly from one of our Leukodystrophy patients!
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*** September is Leukodystrophy Awareness Month ***
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National H-abc/Tubb4a Awareness Day
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First clinical trial for H-abc/Tubb4a Leukodystrophy
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New Scientific Advisor
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Remembering Alice
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Updates on Preclinical Trial Readiness
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Mira's Story
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How Microtubules work and what causes the disruption in the Tubb4a gene causing H-abc/Tubb4A related leukodystrophy