What is the AADC Connects patient registry?
AADC Connects is a registry to collect information about people with AADC deficiency and their experiences with the disease. By providing researchers with a database of detailed medical information about people with a specific disease, registries have proven to be a valuable tool in better understanding of rare diseases.
What are the goals of AADC Connects?
The ultimate goal of AADC Connects is to speed the development of new treatments for people with AADC deficiency. The more we understand about the disease and the more people who participate in research and clinical trials, the closer we can get to finding new treatments.
What does participating in AADC Connects involve?
If you join AADC Connects patient registry, you will be asked to complete a questionnaire about experiences of the person with AADC deficiency, as well as to share medical records that describe diagnosis, treatments, symptoms, and changes in the disease over time. You may occasionally be asked additional survey questions, and to ensure the accuracy of the Registry, you will be asked to update your information a few times a year.
Who has access to AADC Connects?
The broader medical community, including researchers, physicians, patient advocacy groups, and companies engaged in research, can request access to AADC Connects. All information in the Registry is de-identified, meaning it has been stripped of information that could be used to identify you. As a participant, you will have immediate access to the pool of de-identified survey answers.
Will my information be kept confidential?
Yes. When you submit your information to AADC Connects it will be stored and maintained in a secure, password-protected database. Third parties will not be given access to any identifiable patient information, unless you specifically agree to be contacted by them. The Registry adheres to all applicable national, state, and local personal health data protection standards.
Can I withdraw from AADC Connects if I change my mind?
Yes. Participation in this patient registry is entirely up to you. You can withdraw from the Registry at any point for any reason.
How will I learn about clinical trials?
Researchers cannot contact you directly. The Invitae PIN coordinator will send you information about a clinical trial by email. If you are interested, we will explain how to get involved.
Does joining AADC Connects guarantee a spot in a clinical trial?
Joining AADC Connects does not guarantee a spot in a clinical trial because every trial has specific participant criteria that must be met. The benefits of being part of a research-ready community are that it is easier to learn about trial opportunities and to identify patients who are likely to fit the clinical trial criteria.
Are there additional resources available?
Here are links to additional resources that you may find useful for families affected by AADC deficiency, or if would like to learn more about participating in clinical trials.
Connect With Patient Advocacy Groups
AADC Research Trust
Disease Information
NIH
Pediatric neurotransmitter diseases association
Search for Clinical Trials
Clinicaltrials.gov
Rare disease groups
NORD
