2026 H-ABC/TUBB4A Family Conference

đź“Ś Save the Date: May 23, 2026 đź“Ś Save the Date: May 23, 2026

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📌 Save the Date: May 23, 2026 📌 Save the Date: May 23, 2026 〰️

2026 H-ABC / TUBB4A Family Conference

9:00AM - 2:00PM

A Day of Progress, Insight, and Community

The Foundation to Fight H-ABC is proud to sponsor the 2026 H-ABC / TUBB4A Family Conference, hosted by the Leukodystrophy Center at Children’s Hospital of Philadelphia. This virtual event will be held on May 23, 2026, bringing together families, clinicians, researchers, and advocates from across the H-ABC community.

No advance registration is required for this event.
It will be live-streamed at ‍ ‍



www.theglia.org/2026tubb4aconference

Designed first and foremost for families living with H-ABC / TUBB4A related leukodystrophy, the conference offers a meaningful opportunity to hear directly from those advancing research and care, while staying connected to a community that understands the journey. This event will feature presentation by some of the world’s leading clinicians and scientists for TUBB4A-related leukodystrophy, along with biotechnology and pharmaceutical companies working to develop therapies, and advocacy leaders working to raise awareness and funding for tomorrow’s discoveries. 

The event will begin at 9:00AM Eastern Daylight Time. A detailed agenda will be announced at the start of the event; however, attendees should plan on approximately 3-4 hours of presentations followed by an hour-long discussion session during which presenters will make themselves available to take questions from attendees, who will be able to submit these via chat functionality throughout the event. While there may not be sufficient time to answer all questions, the organizers and presenters will do their best to address as many as possible.

This video shares perspectives from families and highlights from the first Leukodystrophy Center Family Conference held at Children’s Hospital of Philadelphia in 2018.

What to Expect

The program reflects both the depth of ongoing research and the growing momentum across the field:

Clinical Research Updates

  • Anjana Seveagamoorthy, MBBS, MPH (Children’s Hospital of Philadelphia) – Natural History Study Updates

  • Francesco Gavazzi, MD, PhD (Children’s Hospital of Philadelphia) – Study Outcome Selection and Validation

  • Giulia Porcari, MD (Children’s Hospital of Philadelphia) – Advancing Clinical Trials Across the Phenotypic Spectrum

Preclinical Advances

  • Sunetra Sase, PhD (Children’s Hospital of Philadelphia) – TUBB4A Animal Models

  • Guangping Gao, PhD (UMass Chan Medical School) – AAV Development

Therapeutic Development

  • Uwe Meya, MD (Synaptix Bio) – Commercialized ASO

  • Laurence Mignon, PhD (N-Lorem Foundation) N-1 ASO

Advocacy and Community Updates
Representatives from global H-ABC/TUBB4A advocacy organizations, including:

  • Foundation to Fight H-ABC/TUBB4A (United States)

  • H-ABC Foundation (United Kingdom)

  • FundaciĂłn TUBB4A (Spain)

  • Kinslow TUBB4A Foundation (United States)

Q&A Session

  • All Scientific Presenters

  • Advocacy Representatives

Why This Matters

Progress in rare disease is built step by step—through research, collaboration, and the shared commitment of families and experts alike. This conference reflects that steady advancement, offering both clarity on where things stand today and perspective on what lies ahead.

Just as importantly, it reinforces something equally vital: no family walks this path alone.

Recordings of all presentations will be posted to this link in the week following the meeting, so please don’t worry if you cannot attend live or need to step away for a portion of the meeting; you will be able to rewatch the presentations at your leisure. The discussion session will not be recorded to promote more transparent interactions and to protect the privacy of our attendees.