Illustrative estimate only - not verified by this sponsor. Contact the study team for actual compensation.
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful functional improvement. This has been a particular challenge in rare and slowly progressive diseases such as Duchenne Muscular Dystrophy (DMD). The investigators hypothesize that urinary N-terminal fragment of titin (NTFT) corresponding to activity level/intensity will define a high-precision, non-invasive biomarker of systemic muscle injury to enable serial measurements of efficacy and safety in the clinical investigation of gene therapy for DMD and other myopathies. This should provide a valuable exploratory, secondary and eventually primary outcome measure of therapeutic efficacy to minimize the enrollment size in informative early phase and pivotal clinical trials.
What happens when you apply
Reach out via phone or email to express interest
Brief call to discuss your health history
Medical screening at the research site
Begin your journey in the study
DMD/BMD Subject Inclusion/Exclusion Criteria Inclusion Criteria: 1. Ambulatory at screening 2. Genetically confirmed diagnosis of DMD/BMD 3. Parental/guardian permission (informed consent) for children. Child assent will also be obtained from patients ages 7 years old and older and deemed by the investigator to be neurodevelopmentally appropriate 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion Criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent Healthy Control Subject Inclusion/Exclusion Criteria Inclusion criteria: 1. Healthy children without DMD, BMD, or other significant chronic medical disease 2. Ambulatory at Screening, defined as able to walk independently without assistive devices 3. Parental/guardian permission (informed consent). Child assent will also be obtained from patients aged 7 years and older and deemed by the investigator to be neurodevelopmentally appropriate. 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent
imaguineapig pulls live data from ClinicalTrials.gov (NIH/NLM).Illustrative estimate only - not verifiedPay estimates are approximate ranges based on study type and are not confirmed by sponsors — actual compensation may differ. Eligibility indicators use limited criteria (age, sex) only. We do not provide medical advice. Always contact the study team directly to confirm compensation, full eligibility, and risks before enrolling.