CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).
Individuals can participate through the CureDuchenne Link™ application (accessible via mobile device or web interface) and receive communications about research opportunities and community programs. Participation may be done using virtual methods, at a project site, and/or at community events nationwide. All collected information will be stored in a secure, HIPAA-compliant data warehouse for approved researchers to use for studies relevant to DMD, BMD and other neuromuscular disorders. Combining health and outcomes data with biospecimens provides an impactful solution and novel resource for researchers, allowing for effective translational research.
Study Type
OBSERVATIONAL
Enrollment
240
Arkansas Children's Hospital
Little Rock, Arkansas, United States
CureDuchenne
Newport Beach, California, United States
Children's Hospital of Orange County
Orange, California, United States
Diagnosis
There is no intervention in this project. Participants will provide documentation to support their diagnosis of Duchenne muscular dystrophy, Becker muscular dystrophy, or a carrier of these mutations
Time frame: Upon study entry
Genetic Mutation
Participants will be asked to provide genetic testing reports confirming their diagnosis, where available, which will be reviewed by a central genetic counselor.
Time frame: Upon study entry or when genetic testing results are available
Functional Status
Self reported data (questionnaire on ambulation and mobility) will be captured
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
North Star Ambulation Assessment (NSAA) Score
Clinically reported NSAA scores will be captured
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
6 Minute Walk Test (6MWT) Score
Clinically reported 6MWT scores will be captured
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Corticosteroid Status
Self reported and clinically reported corticosteroid status (past and present) will be captured
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Cardiac Status
Self reported and clinically reported cardiac status (past and present) will be captured
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Rare Disease Research
Atlanta, Georgia, United States
University of Iowa
Iowa City, Iowa, United States
Kansas University Clinical Research Center
Fairway, Kansas, United States
Corewell Health
Grand Rapids, Michigan, United States
Rare Disease Research Center
Hillsborough, North Carolina, United States
Penn State Health
Hershey, Pennsylvania, United States
Neurology Rare Disease Center
Denton, Texas, United States
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Respiratory Status
Self reported and clinically reported respiratory status (past and present) will be captured
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years