Leukodystrophy Center Clinical and Research Opportunities
The Leukodystrophy Center has set aside slots for clinical and research evaluations in the days leading up to the conference. Additional details regarding each type of visit are summarized on the next page.
Individuals interested in scheduling clinical and/or research evaluations at the Children’s Hospital of Philadelphia (CHOP) in the context of their visit should contact the Leukodystrophy Center’s coordinators by filling out an Online Referral Survey via the link below.
CHOP Leukodystrophy Center Referral Survey
In the unlikely event that demand for clinical and/or research evaluations exceeds the number of slots available, priority may be given to individuals who have not previously been evaluated at CHOP, as well as individuals who are traveling significant distances to attend the 2023 4H Family Conference.
Leukodystrophy Center Clinical Appointments
Individuals and families interested in obtaining formal medical guidance and recommendations from the Leukodystrophy Center’s multidisciplinary team of providers, including neurologists, genetic counselors, physical therapists, etc. will need to schedule a clinical appointment in their outpatient clinic at the Buerger Center for Advanced Pediatric Care, located within the Children’s Hospital of Philadelphia’s main campus. Clinical appointments will either be billed to insurance or paid for out of pocket, though individuals and families may apply separately for financial support through the Yaya Foundation. Please contact Ben Smith at email@example.com directly for information regarding potential financial assistance.
The Leukodystrophy Center has 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 internationally.
Leukodystrophy Center Research Assessments
The Leukodystrophy Center hosts one of the world’s largest leukodystrophy-specific data and sample repositories – formally 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 4H. Individuals and families who express interest in research participation while completing the Online Referral Survey will have an opportunity to speak with an experienced research coordinator to learn more about eligibility requirements, study activities, etc., and to schedule research assessments in the context of their visit to Philadelphia.
Please note that our research providers are not permitted to provide medical guidance based on a research assessment alone – this would require scheduling a clinical appointment, as discussed above. Any assessments or other activities performed explicitly for research purposes will be paid for by the program’s research funds.
If you have any questions regarding the logistics of scheduling a clinical appointment and/or participating in research assessments, you may contact the Leukodystrophy Center’s Administrative Manager, Omar Sherbini, MPH, at (215) 590-3068 or firstname.lastname@example.org.