The Leukodystrophy Center at Children’s Hospital of Philadelphia is pleased to host the 2026 TUBB4A Family Conference on Saturday, May 23, 2026. 

Organized in partnership with several of the leading disease-specific patient advocacy organizations - Foundation to Fight H-ABC (USA), Fundación Leucodistrofia TUBB4A (Spain), H-ABC Foundation (UK), Kinslow TUBB4A Foundation (USA) - this event seeks to bring together families, clinicians, and researchers to share the latest developments in TUBB4A-related leukodystrophy. 

Tentative Schedule

 In the days leading up to Saturday’s conference, families will have opportunities to participate in clinical evaluations and research assessments. Please see below for a general schedule of events; detailed information (including locations and timing) will be made available in the months leading up to the event.

Clinical Evaluations

Families interested in obtaining formal medical guidance and recommendations from the Leukodystrophy Center’s multidisciplinary clinical team, which includes highly specialized neurologists, genetic counselors, physical therapists, etc. will need to schedule a clinical evaluation. 

The clinical evaluation is separate from the registration process for the conference.

Appointments will be available on Thursday, May 21, 2026 and Friday, May 22, 2026. Depending on demand, additional slots may be opened on Wednesday, May 20, 2026. Clinical evaluations must be billed to insurance or paid for out-of-pocket. Families who are unable to cover the associated expenses independently may apply for financial support through one of our advocacy partners using the financial assistance form linked below. 

The Leukodystrophy Center has dedicated coordinators available to assist with scheduling, record collection, insurance authorization, etc., and will work closely with the hospital’s Global Patient Services (GPS) to generate cost estimates and streamline logistics for families traveling from abroad.

Research Assessments

The Leukodystrophy Center hosts one of the world’s largest leukodystrophy-specific research repositories - known as the Myelin Disorders Biorepository Project (MDBP) - that has supported key leukodystrophy discoveries over the past two decades and continues to serve as an essential resource for patients and families, clinicians, and research collaborators alike.

It also serves as the umbrella research protocol for a series of international natural history projects being coordinated by the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), including one for TUBB4A-related leukodystrophy. Individuals who have already consented to the MDBP or may be interested in doing so prior to the conference will be invited to schedule a research assessment in the context of their visit to Philadelphia. 

There is no cost associated with research assessments; however, please note that our research providers will not be able to provide formal medical guidance based on these assessments alone. Families interested in obtaining clinical guidance must schedule a comprehensive evaluation with the Leukodystrophy Center’s multidisciplinary clinical team, as discussed above. 

Registration Deadlines

Families interested in scheduling clinical evaluations and/or research assessments must complete the registration form no later than February 14, 2026. Similarly, families interested in requesting financial assistance to offset costs associated with accommodations, clinical care, travel, etc. must submit this Financial Assistance Form to Omar Sherbini no later than February 14, 2026.

Families who do not plan to participate in clinical or research activities and wish to simply attend the conference may continue to register after this deadline. 

Questions

If you have any questions, please feel free to contact the Leukodystrophy Center's Administrative Director, Omar Sherbini, MPH, at sherbinio@chop.edu or 215-590-3068. We hope to see you in Philadelphia!