Tag Archives: #4HLFamilies

Launching the 4H Leukodystrophy Data Collection Program

Register for Info Session to Learn More

Collecting data is an essential first step on a rare disease research journey and an important way for patients and their caregivers to contribute to advancing care for their communities.

On a recent family call, Dr. Nicole Wolf, one of the world’s leading clinician scientists of 4H/POLR3-related Leukodystrophy spoke to the importance of data collection. She mentioned that this data informs the natural history of a disease and can be helpful in several ways, including:

  • Counseling families what to expect of the disease
  • Understanding the basic disease mechanisms
  • Evaluating the effect of treatments
  • Quickly identifying possible clinical trial candidates

Launching on June 21st, the Yaya Foundation is excited to announce a new 4H/POLR3-related Leukodystrophy Data Collection Program (also called a disease registry). The Foundation has partnered with leading rare disease data collection experts RARE-X.

To learn more about the Data Collection effort and benefits, please register for an upcoming Information Session on June 16th.

To hear more from Dr. Wolf on the importance of data collection, you are invited to watch her presentation on this topic: