On May 16th, the Yaya Foundation invited a very special guest to join the #4HLFamilies call – Dr. Nicole Wolf of the Amsterdam Leukodystrophy Center and the Amsterdam University Medical Center. Dr. Wolf is one of the world’s leading clinician scientists of 4H/POLR3-related Leukodystrophy.

Several important topics were presented including the history of 4H Leukodystrophy, how this rare disease is described and diagnosed, both typical and atypical clinical course, and current research in 4H Leukodystrophy. Dr. Wolf also described the critical importance of data collection efforts, or disease registries, in further understanding rare diseases, designing clinical trials and identifying possible clinical trial candidates. The families also discussed topics important to them, including the Covid vaccine.