The MDF went to Washington, DC, last week to represent the DM community and participate in the Rare Disease Legislative Advocates Conference and Lobby Day.
You can help the research community better understand myotonic dystrophy (DM) and improve the lives of those living with DM by joining the new Myotonic Dystrophy Family Registry (MDFR). Click here to visit the MDFR website.
This new Registry -- launching on Rare Disease Day, February 28th, 2013 -- is unique in that it will not only help researchers find new, effective treatments and identify possible participants for upcoming clinical trials and research studies, but will also allow anyone who is registered to have access to the collected data, from academic and pharmaceutical researchers to individuals and families living with DM. The goal is to give all DM stakeholders, including family members, a better understanding of the disease, the DM community, and current research and advocacy efforts.
By joining the MDFR, you can aid researchers, pharmaceutical companies and other professionals seeking to:
- Identify participants for clinicial trials and research studies
- Develop new, effective treatments for those living with DM
- Advocate on behalf of the DM community with decision makers in Washington, D.C. and elsewhere
Because you will have access to de-identified information in the MDFR, you can learn more about DM, how it's experienced by other community members, and what the broader community looks like. This includes:
- Information regarding clinical trials and studies for which you or members of your family may be eligible
- Where Registry participants are located
- What symptoms are most and least prevalent
- How other community members manage living with DM
- How DM has impacted other families living with this disease
Your participation is voluntary and your individual information will be kept completely confidential. You can opt out of the Registry at any time and all of the data you provide will be entirely de-identified (anonymous). This means that if you're potentially eligible for a clinical trial or research study based on your Registry data, researchers will contact us first. We will follow up with you and, if you would like to learn more, will connect you with the interested research organization. You will never be contacted by anyone but MDFR staff.
The more participants we have in the MDFR, the more helpful the Registry wil be, and size is critically important to helping support clinical trial and research study recruitment. Please share our news with others in the DM community and help us build a large, rich database that will help accelerate efforts to treat and cure this disease.
Starting February 28th, 2013, click here to go to the Myotonic Dystrophy Family Registry website, to learn more and sign up.
And if you haven't already done so, please join the National DM & FSHD Registry housed at the University of Rochester, which will continue to develop critically important natural history, disease progression and other myotonic dystrophy data to promote long-term understanding and discovery. Click here to learn more.