We are funding a natural history study for DRPLA!

by | Jun 30, 2022 | News Articles

The DRPLA Natural History and Biomarkers Study has three main objectives:

  1. Researchers would like to characterize the natural history of DRPLA, that is to say, how subjects with DRPLA change over time.
  2. This study will identify genetic factors and biomarkers that could predict disease progression. A biomarker is any variable/characteristic that informs of physiological processes, pathological processes or response/effects of a drug.
  3. This study will provide useful information to support the design and conduct of clinical trials in the future.  

This study will collect health information from DRPLA individuals over three years to understand how this condition develops over time. The study also recruits participants without DRPLA, so that they are able to compare how individuals with DRPLA differ from those without. Participating in this study will involve annual appointments at the clinics where the neurologist will do some clinical assessments (e.g. ataxia scales, brain MRI, blood collection, etc.).

This study has sites in the United Kingdom (London), the United States (New York and North Carolina) and Japan. Although participants from anywhere in the world could also be recruited.

The North Carolina site has started recruitment and you can find the recruitment flyer here. The other sites will follow shortly.

For more information, please contact the Study Coordinator, Silvia Prades, at silvia.prades@curedrpla.org