CureDRPLA Global Patient Registry for Individuals With Dentatorubral-pallidoluysian Atrophy (DRPLA)
CureDRPLA
Summary
The objective of the CureDRPLA Global Patient Registry is to establish a longitudinal database of patient-reported data on individuals affected with Dentatorubral-pallidoluysian atrophy (DRPLA) from anywhere in the world. The CureDRPLA Global Patient Registry will address patient needs by: * Expanding patient engagement by documenting quality of life outcomes. * Providing anonymized data to the DRPLA research community on patient experience with the disease and priorities for treatment. * Connecting DRPLA patients with opportunities to participate in clinical research.
Description
The CureDRPLA Global Patient Registry aims to collect data from Dentatorubral-pallidoluysian atrophy (DRPLA) patients worldwide to identify a well-characterized cohort for participation in retrospective and prospective research. Participants will be required to read and sign an IRB-approved informed consent document prior to accessing questionnaires within the Registry. The Registry is solely for patients diagnosed with DRPLA. After informed consent is given, and assent for those age 12 to the age of consent, participants will be asked to complete a series of questionnaires to include informa…
Eligibility
- Age range
- 0–100 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Individuals of all ages with a self-reported diagnosis of DRPLA. * Understand and sign the informed consent form (IFC). Participants who lack the capacity to consent (e.g. cognitively impaired individuals) will require consent from the legal authorized representative, and the assent of the subject will be obtained to the extent compatible with their capacity. Participants at age 12 to age of consent will require assent along with the consent of their parent or legal guardian. Exclusion Criteria: * Ataxia conditions other than DRPLA. * Failure to sign the IFC (and assen…
Location
- CureDRPLANew York, New York