Parent Project MD has developed a patient/family registry for Duchenne and Becker MD with the name DuchenneConnect. Are you connected? The process is easy and the organizers are working to improve the site making it simpler to update information. There are just over one thousand individuals participating which is a fraction of those living in the US with D/BMD. We need to encourage all families to join and be part of this excellent resource.
The site is more than a collection of information for research. The site is designed to provide useful information for families affected by D/BMD. Upon completing a Profile, families can print a report that can be taken their doctor's appointments. The Profile Report lists specific aspects of an individual's health. Bringing the report to a doctor's visit provides an opportunity to obtain information which a family may not have access and a physician can verify that the data is correct. The site has posted clinical care recommendations based on expert recommendations and linked to the TREAT NMD care site. These recommendations may also be printed and taken to the doctor's visit to discuss areas of concern or when seeking additional information about a certain health topic. When families obtain more up to date information changing the profile is quick and easy. This is important to keep the registry current.
Families can research ongoing clinical trials that are actively recruiting or will soon recruit participants. Families are able to search the site and may learn how other individuals with DMD compare based on age and other information. As more participants are added we may develop a better sense of how individuals are doing based on care, medications, supplements and mutation type.
This data base is a tool researchers are able to use to learn more about D/BMD. Clinicians and researchers are able to search de-identified information based on age, mutation type and other factors. As the data base grows it will provide researchers a sense of the numbers of individual who may be available to participate in a clinical trial. No one will be contacted by a research, yet those expressing an interest in participating in research trials may be contacted by the DuchenneConnect Coordinator if they meet eligibility criteria.
While most clinical trials are thought to be a few years away it is still very important to participate. Researchers will need participants for trials and they cannot begin searching for families willing to participate when they are ready to begin as it will significantly delay the process. A researcher involved in a recent clinical trial for another muscular dystrophy expressed frustration when he realized they needed to find participants as no data base existed. The researcher said if they realized the efforts needed they may not have pursued the project. The DuchenneConnect Registry is designed to stay ahead of that curve.
I realize families are busy and time is always in short supply. Digging through medical records to gather test results is a chore, but it is something that can be done as time allows. If a mutation is not known the DuchenneConnect Coordinator can help families choose the type of testing needed. The Coordinator can help families needing help completing the Profile of with understanding results of genetic tests they may already have. This is a well supported site. Families should be encouraged to participate in this wonderful effort that will be a vital link between researchers, clinicians and families.
Have you completed a profile? Are you Connected? Please look into participating today!