A decentralized platform connecting researchers to coded data and samples from people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) was presented in a poster at the National Organization for Rare Disorders (NORD) Breakthrough Summit 2021.
The platform, called CureDuchenne Link, is a data hub that connects the information provided by DMD and BMD communities directly to researchers.
It allows participants residing anywhere in the United States to share their information and samples. Qualified researchers can then access the coded data and biosamples without any personally identifiable information attached to them.
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The platform was launched in early 2020 as a pilot study with the participation of 18 people from 14 households, all of whom rated their overall experience as good or excellent and said that they were very likely to recommend participation. They said that the surveys were comprehensible but not overwhelming. “This is key to increasing participant retention,” Jamie Gullikson, MPH, MBS, clinical research coordinator at CureDuchenne, and the coauthors of the poster wrote.
The platform launched for nationwide enrollment in the United States in July 2021 and reached 49 participants from 43 households in 25 states within 5 days of the launch. Of these, almost all (97.1%) agreed to provide medical records, and the majority (87.8%) also agreed to provide at least 1 type of sample.
The researchers also reported that beginning in 2022, the platform will expand to include archived muscle tissue and facilitate the distribution of coded data and samples to researchers focusing on muscular dystrophy.
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CureDuchenne is a nonprofit organization in Newport Beach, California with a mission to accelerate research to find the cure for DMD and improve care, as well as to empower the Duchenne community.
CureDuchenne Link is the first comprehensive, widely accessible database of DMD and BMD biological samples with integrated clinical data in the United States.
Gullikson J, Kemp J, Miller D; CureDuchenne. Novel model for decentralized data and biosample repository for Duchenne and Becker muscular dystrophy. Poster presented at: NORD Breakthrough Summit 2021; October 18-19, 2021; Virtual.