Patient advocacy groups like the Yaya Foundation can play many roles – educating, raising funds and driving research strategy, advocating for, and providing support services to our families. One of the important support services comes in the form of bi-monthly #4HLFamilies Zoom calls – event details can be found on our Facebook page.
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.
The Yaya Foundation is incredibly grateful to Dr. Wolf for her valuable time, insight and care for families affected by 4H Leukodystrophy. You can watch the discussion here:
“It is so nice to have Dr. Wolf share time with us, she diagnosed my daughter 8 years ago and was part of the team that discovered the syndrome. Without her, hundreds of families would still be searching for answers. Dr. Wolf gave us hope that some day there will be treatments or even a cure for 4HL.”
Finally, thank you to our families affected by 4H for actively engaging in the hosted #4HLFamilies calls.
The Yaya Foundation is committed to hosting family calls and events that are relevant for everyone, including those newly diagnosed. Please join us for the next call on Sunday, July 18th.