This Registry has been created specifically for individuals who have a diagnosis of Duchenne or Becker muscular dystrophy, and for carriers of Duchenne or Becker.
You must be 18 years or older to register, but parents and guardians may register on behalf of children and teens with Duchenne/Becker. Individuals who live with or care for adults with Duchenne/Becker may also help with Registry participation by answering questions on their behalf. However, each registrant can only have one account in the Registry.
This Registry was designed for individuals in the United States and is only available in US English, but participants from any country in the world are welcome to participate. We have registrants from over 100 countries. If you live outside the US, click here to see if your country has a registry. If so, we encourage you to participate in your country’s registry as well as The Duchenne Registry.
Participation in the Registry is completely voluntary. It is your choice to participate. You may also stop participating for any reason and at any time. If you decide not to participate or if you decide later to withdraw from the Registry, we will not penalize you or ask you for an explanation. However, we appreciate your feedback so that we can continue to improve the Registry.
If you decide to participate now but later wish to withdraw, you will need to contact The Duchenne Registry Coordinators and we will no longer contact you. If you withdraw, you will be able to choose whether we can continue to use your data that is already in our Registry, or whether you want us to remove all of your data from our Registry. No matter which option you choose, we cannot retrieve data that has already been used in research and/or provided to investigators before your decision to withdraw.
The goal of this Registry is to make the information you provide searchable and widely usable, while protecting your identity. Clinicians, researchers and pharmaceutical companies who access the Registry data can better understand Duchenne and Becker. The Registry data can also be used to make the research and clinical trial process faster and more efficient. In addition, the Registry also offers you access to information regarding clinical trials and research studies that may be a good fit for you or your child.
To better understand your health and your daily experience living with Duchenne/Becker, we will ask you to respond to several surveys. We will also ask you to share a copy of your genetic test report. You can decide how much information you wish to share. However, the more data we have, the more we can share with researchers and the better we can tailor information to your specific needs.
The Registry will contact you, usually at least once a month, through app notifications and emails. These will be requests to complete a survey, educational newsletters, or notifications about new research studies and clinical trials, or upcoming activities such as webinars and conferences.
Participating in a registry comes with a small risk that information about you will be disclosed to someone who is not authorized to see it. The Registry team has taken the necessary steps to reduce this risk and protect your information.
The data collected and compiled by this Registry belongs to the registrants. PPMD is the guardian of this data, but PPMD staff who are not on the core Registry team do not have access to your Registry data. Only the Registry Director and the Registry Coordinators have access to this data.
Your name and contact information will never be given to anyone without your permission. The Duchenne Registry is deeply committed to protecting your privacy and identity, and will use every available measure to ensure the security of your personal information.
The Registry is hosted by THREAD Research, a leader in virtual research platforms and mobile apps. THREAD Research complies with important research and privacy regulations for protecting patient data in research.
THREAD Research will replace your name and other personal information with a code. The coded data will be encrypted and stored on a secure Cloud server. Your name and any information that directly identifies you are not included in any records or samples that are reviewed and/or copied, or sent to researchers. Instead of your name, a unique code number is used. Your coded data will be stored for a maximum of 80 years after the end of this study. After that your data will be deleted.
In order to help advance research for Duchenne, we will share your de-identified data with eligible researchers around the world. De-identified means that personally identifying information, such as names and addresses, has been removed. The Registry team carefully reviews all requests for data and determines the validity and importance to the community. If the request for data is approved, the Registry staff provides counts and/or de-identified data exports to the researcher.
A new addition starting in 2019 will be the ability to pull all data in The Duchenne Registry into the Duchenne Outcomes Research Interchange, a data hub that combines data from multiple sources in an effort to accelerate research for Duchenne and promote collaboration among researchers and clinicians. Electronic health record (EHR) data will be pulled into the Interchange from several hospitals across the United States, so if you are seen at one of the participating hospitals, your EHR data will be combined with your Duchenne Registry data in the Interchange.
The EHR items to be shared with the Interchange include the FHIR US core data elements, also known as the Common Clinical Data Set, including but not limited to basic demographics, problem lists, medications, vitals, laboratory results and procedures.
In addition to sharing data with the Interchange, some of your information will be shared with other research groups. For example, a subset of de-identified data from each registrant will be shared with the TREAT-NMD International Neuromuscular Network in order to enhance global knowledge of Duchenne.
We also share all of the de-identified data with the Duchenne Regulatory Science Consortium (D-RSC) at the Critical Path Institute. The D-RSC team is pooling data from multiple sources to develop a Duchenne disease progression model. They have done similar work with other rare and common disorders.
If you have any questions about how your data will be used, please email coordinator@duchenneregistry.org or call 888-520-8675.
The time to complete surveys will vary, and we try to keep them as short and simple as possible. We want your Registry experience to be easy and not burdensome for you or your family. If all surveys are answered at the same time, it could take approximately 30 minutes to complete. However, not all surveys need to be completed at the same time.
You do not usually receive compensation (money or gifts) for participating in The Duchenne Registry. However, for some IRB-approved research studies, reasonable compensation may be provided to families for time spent completing additional questionnaires. In addition, we often conduct raffles in which registrants who complete a particular survey may be eligible to win a non-monetary prize or a gift card.
Benefits to participating in The Duchenne Registry include helping to speed research and advance treatments in Duchenne and Becker by sharing your data; belonging to a global research network of Duchenne and Becker families who are acting as citizen scientists; having access to Registry staff (including certified genetic counselors) who can answer your questions; and receiving notifications of clinical trials and research studies that may be a good fit for you or your child.
The Registry works with researchers who have clinical trials or research studies and need volunteers. We never provide your name or contact information without your permission. Instead, we give you the power to decide whether to contact researchers to learn about their studies. We search the Registry to see who appears to match the main inclusion criteria for a study and then email you information about the study. If you are interested, you make contact with the researchers, who will determine whether you or your child is eligible to participate and will answer any questions you may have about the research.
Participating in The Duchenne Registry entails a small risk of unauthorized use of your data. We take great care to protect your information; however there is a slight risk of loss of privacy. This is a low risk because we separate your personal information (such as your name, address and phone number) from the rest of your Registry data in order to respect your privacy. In the event of unauthorized access, it is sometimes possible to re-identify an individual. This risk, while low, should be considered before enrolling.
If such an unauthorized use occurs, you will be immediately notified. If there is a breach in the security of THREAD Research, then all participants will be notified of the steps required to repair the breach and prevent its recurrence.
In addition to this risk to your privacy, there is also a small risk of psychological discomfort in answering questions about your experience with Duchenne or Becker. You are not required to answer these questions if doing so causes you discomfort.
Participation in the Registry entails no physical risks for you or your family.
The Duchenne Registry Coordinators are available to answer your questions and assist you with completing your Registry account. Email coordinator@duchenneregistry.org or call 888-520-8675.
If you have questions regarding the consent process or the Registry’s IRB-approval, please contact the Geisinger Health System IRB at IRB@geisinger.edu or call 844-542-3299.