What is DRPLA?
Dentatorubral-pallidoluysian atrophy, commonly known as DRPLA, is a progressive brain disorder that affects coordination, balance, speech, and causes involuntary movements, emotional problems, and a decline in thinking ability. The average age of onset of DRPLA is 30 years, but this condition can appear anytime from infancy to adulthood. The symptoms and progression vary depending on the age of onset.
What is CureDRPLA?
CureDRPLA is a US based non-profit founded by Paul and Andrea Compton. Paul and Andrea have a son that was diagnosed with DRPLA in August 2018. The mission of CureDRPLA is to connect families, physicians and scientific investigators to further DRPLA research and work towards a treatment for DRPLA. This webpage was created to find others that have this condition and are interested in finding a cure.
The CureDRPLA Global Patient Registry is a worldwide registry of DRPLA patients.
This registry gathers the demographic and clinical information on DRPLA patients from across the globe. By collecting information on all DRPLA patients, the CureDRPLA Global Patient Registry will be a powerful resource for research and will enhance opportunities for treatment development.
Patient & Caregiver
Click below to find out what CureDRPLA has done and what we are currently doing to find to a treatment for DRPLA. You will also find online resources and support organizations for DRPLA as well as YouTube channels specifically focused on DRPLA.
Click below to find out what CureDRPLA has done and what we are currently doing to find a treatment for DRPLA. You will also find online resources and support organizations for DRPLA as well as YouTube channels specifically focused on DRPLA.
CureDRPLA is investing in a number of projects in hopes of rapidly developing novel therapeutics for DRPLA. Information on funding opportunities, a list of our preclinical projects as well as other efforts can be found here.
Support NAF in asking the FDA to incorporate regulatory flexibility when evaluating drug applications for rare diseases
The FDA, the US regulatory agency for new medicines, declined to review the documents for a potential therapy for SCA3 (type of ataxia) developed by Biohaven. The criteria for approving a drug often rely on standards established for more prevalent conditions, making...
If you ever asked yourself this, the map we have created will come in handy. We coloured in orange those countries in which at least one person with DRPLA has been identified – 35 countries in total! We have gathered this information from scientific articles and...
On February 22nd, I organised a meet up for families affected by DRPLA who are based in Wales. I travelled to Cardiff with Dr Hector Garcia-Moreno and Ola Volhin, who work at University College London on the DRPLA Natural History and Biomarkers Study. We met with...