As the Rett Syndrome Research Trust (RSRT) prepares to launch a global database of caregiver-provided information called The Rett Syndrome Global Registry, it is seeking families to participate in a pilot program to test its objectives.
The goal in launching the registry is threefold: first, to provide a way for researchers and physicians to easily tap into the shared knowledge and day-to-day experiences of parents and other caregivers of people with Rett syndrome; second, to provide those caretakers with tools to better manage their child’s needs; and finally, to advance research toward effective therapies.
In preparation for the planned launch early next year, the RSRT is asking families to provide their input and help test the registry in a pilot program called Registry Pioneer. Interested caregivers can sign up here. According to RSRT, the Pioneer program will enable the registry to start with a foundational data set and be of immediate use.
Information found in the global registry would include children’s symptoms, therapies, and the state of their skills.
Parents and caregivers would be able to track and graph these features on smartphones and other hand-held devices. This information can be shared with other parents in the global community, with doctors who can make reporting a child’s history more accurate, and with pharmaceutical companies to design more efficient clinical trials.
The registry also may make it easier for researchers and clinicians to identify patterns and frequencies of symptoms in Rett patients around the world.
“Parents and caregivers are most assuredly experts on Rett Syndrome, and in particular, they’re experts on their own children,” the RSRT wrote on its website. “No one knows your child’s symptoms, challenges, strengths, subtleties, and routines better than you.”
Given the small number of patients, recruiting participants into clinical trials in rare diseases is frequently a challenge and a bottleneck to conducting successful studies. Online and app-based tools to gather patient information are increasingly seen as a way to reach underserved populations.
Large registries give researchers more data to work with when looking for eased symptoms and improved skills that matter most to patients and their families.
The RSRT hopes it also will provide crucial insights into existing research questions and point the way toward asking new ones.
Since the RSRT was founded in 2008, the charitable organization has awarded $66 million to research initiatives aimed at treating and curing Rett.
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