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The Duchenne Registry (Previously DuchenneConnect)
study id #: NCT02069756
condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
DuchenneConnect is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of DuchenneConnect is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, DuchenneConnect is a valuable resource for clinicians and researchers in academia and industry, allowing access to a de-identified, aggregate dataset provided by patients and their families—information that is vital to advances in the care and treatment of Duchenne. DuchenneConnect is a member of PCORnet, the National Patient-Centered Clinical Research Network.
mechanism of action: No pharmaceutical intervention
last updated: November 22, 2018
start date: October 2007
estimated completion: October 2027
size / enrollment: 10000
DuchenneConnect was created in 2007 by Parent Project Muscular Dystrophy (PPMD), with assistance from the NIH, the CDC, and Emory Genetics. In early 2011, PPMD alone began financing the registry's operation and maintenance, and is the sole guardian of DuchenneConnect and its material.
Questions may be addressed to the DuchenneConnect Coordinators at telephone 888-520-8675 or firstname.lastname@example.org. The DuchenneConnect Coordinators are certified genetic counselors who are available to answer questions regarding the registration process, genetic testing and counseling, and clinical trials and research studies.
- Genetic mutation [ Time Frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime. ]
Genetic mutation data is collected by patient report and verified by curation/review of genetic test report when provided. Genetic test report is requested for each registrant and is required for participation in global DMD (TREAT-NMD) registry.
- Ambulation status [ Time Frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime. ]
Ambulation status is assessed from several questions about mobility, ability to sit and stand, use of assistive devices, and age at full time wheelchair use.
Diagnosis of Duchenne or Becker muscular dystrophy; Manifesting female carriers and asymptomatic female carriers also included in registry.
Diagnosis of any other type of muscular dystrophy (including limb-girdle muscular dystrophy).
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