Foundation to Fight H-ABC

08/14/2026

Today, the Foundation to Fight H-ABC had the opportunity to participate in the 2026 NINDS Nonprofit Forum, hosted by the National Institute of Neurological Disorders and Stroke and the American Brain Coalition.

This year’s theme, “Progress through Partnership,” could not be more relevant to rare disease. For families living with H-ABC/TUBB4A-related leukodystrophy, progress depends on researchers, clinicians, government agencies, industry, nonprofit organizations and patient communities working together.

Forums like this give small rare disease organizations an important opportunity to be part of the broader neurological research conversation, learn where the field is headed, build relationships and make sure the needs of our families are represented.

We continue to look for every opportunity to bring greater attention to H-ABC, strengthen research partnerships and move our community closer to meaningful treatments.

We are grateful to NINDS and the American Brain Coalition for bringing the nonprofit and patient advocacy communities together.

08/14/2026

September is Leukodystrophy awareness month. H-abc is a Leukodystrophy ! Sport one of our T shirts! We have a basic all season T or if you scroll down you’ll see this one shown in the picture. Go to our Store at H-abc.org.

08/13/2026

This week the Foundation joined Rare Across America to advocate for legislative support from The Honorable Chris Van Hollen, United States Senator for Maryland and The Honorable Jamie Raskin, United States Representative for Maryland’s 8th Congressional District for several pieces of legislation that are particularly relevant to the rare disease community:

H.R. 1532 – Scientific EXPERT Act: Would require FDA participation in externally led scientific meetings focused on rare diseases, bringing regulators, researchers, drug developers and patient organizations together to address barriers to treatment development. This is especially important for ultra-rare diseases where traditional clinical trials may not be feasible.

H.R. 6280 – Access to Genetic Counselor Services Act: Would improve access to genetic counseling by allowing qualified genetic counselors to provide and be reimbursed for services through Medicare. This would help families better access and understand genetic testing, diagnoses and inherited conditions.

H.R. 7118 – Genomic Answers for Children’s Health Act: Would improve Medicaid access to whole-genome and whole-exome sequencing for children with suspected genetic or rare diseases, helping reduce the years-long diagnostic journey many families experience.

Of particular importance to H-ABC is H.R. 1532 because ultra-rare diseases need a regulatory pathway that recognizes very small patient populations and allows FDA, researchers, developers and patient organizations to work together early to determine realistic evidence and development requirements.

These are all important efforts to address different pieces of the rare disease journey—from diagnosis and genetic counseling to actually getting treatments developed and approved.

08/04/2026

September is Leukodystrophy Awareness Month and the Foundation is gearing up for our awareness campaign!

If you would like to support us here is how:

Buy and wear this t-shirt during the month. https://www.bonfire.com/leukoawareness-1/

Also we have a repurposed drug candidate to help with the symptoms! We need to do an observational study with several patients to see its impact! If you’d like to help us do a peer to peer fundraiser over the course of the month to help fund this study, please message me!

Michele Levoir Sloan

Buy Foundation to Fight H-ABC Store merchandise that supports Foundation To Fight H-ABC. Featuring Athletic Heather Premium Unisex Tees, professionally printed in the USA.

07/30/2026

One of the biggest challenges in developing treatments for brain disorders isn’t finding a promising therapy, it’s getting that therapy where it’s needed.

This new Nature Reviews Drug Discovery article explains how “brain shuttle” technologies are beginning to change that by helping large biologic medicines cross the blood-brain barrier. It’s an important step forward for many neurological diseases, including rare disorders like H-ABC, where future treatments such as gene therapies, A*Os, and other biologics all depend on reaching the brain.

We’re encouraged to see continued innovation in this area. Progress in treatment isn’t just about developing new therapies. It’s also about solving the delivery challenges that have limited so many promising approaches.

Worth the read:

https://www.nature.com/articles/d41573-026-00121-6

*O

Efforts intensify to ferry large-molecule medicines into the brain, following the first FDA approval of an engineered biologic that overcomes delivery hurdles.

07/19/2026

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07/14/2026

As patient advocates, we spend so much time focused on research, treatments, clinical trials, and access to care. Those things matter tremendously—but so do the people providing that care every single day.

I’m encouraged to see the Child Neurology Foundation launching another Caregiver’s Compass training program starting July 20. This free, therapist-led virtual course is designed specifically for caregivers of children with neurological conditions and provides practical tools for managing stress, navigating grief, strengthening relationships, and building resilience alongside other caregivers who truly understand the journey. I’ve taken the course, it’s wonderful!

For many of us, caregiving isn’t measured in days or months—it’s measured in years and decades. We become nurses, therapists, care coordinators, advocates, researchers, and often the one constant in our loved one’s life. That responsibility comes with tremendous purpose, but it also comes with exhaustion, isolation, and loss that isn’t always visible.

As we continue working to accelerate research for H-ABC/TUBB4A-related leukodystrophy and expand support for the broader rare disease community through Maryland Rare, we also have to invest in caregivers. Supporting caregivers isn’t separate from improving patient care—it is improving patient care.

If you’re caring for a child with a neurological condition, I encourage you to explore this opportunity. And if you’re part of the rare disease ecosystem—as a clinician, researcher, industry partner, or advocate—I hope you’ll continue to recognize that caregiver education and emotional support deserve to be treated as an essential part of care, not an afterthought.

Thank you to the Child Neurology Foundation for continuing to invest in families.



Register here:

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07/08/2026

We feature stories about our community and this one is about Elouise and Camden and, well dolphins!

Connecting and sharing mean the world to both kids affected by a rare disease and also their caregivers!

Enjoy!

As Elouise’s mom, I know that many of the experiences she has are possible only because of a great deal of planning, support, flexibility, and family commitment. Life with H-ABC/TUBB4A related leukodystrophy affects almost every part of our daily life, so even joyful moments often require extra he...

07/07/2026

Meet our volunteers. Thank you for your time, thank you for your hard work, thank you for your commitment to our cause! You all rock!!!

Our volunteers support the mission in many ways, including awareness, education, research support, storytelling, outreach, and community connection.

07/03/2026

If you are a student or are a parent of a student and interested in advocating for a rare disease on campus/school, see the attached and registration link. We would love if you chose our rare disease!!

https://rarediseases.zoom.us/meeting/register/XKG2mlmaS9aPyivi7yTK3Q #/registration