Eligibility criteria are protocol rules, not judgments about a child. Ask the site to pre-screen the exact genetic report, age, function, labs, antibodies and treatment history before making travel plans.
What this means in real life
Trial eligibility criteria protect participants, define the scientific question and make results interpretable. Age windows may be based on disease stage or available safety data. Mutation rules may target a biological mechanism. Walking status and timed-function thresholds create a measurable population. Prior-treatment washouts reduce confounding or interaction risk. These rules are protocol requirements, not judgments about whether a child is “doing well enough.”
Details matter. Age may be calculated on the screening or dosing date; ambulatory status may have a protocol definition; a timed test may need two qualifying results; stable steroid dosing may be required for months; prior gene therapy may be a permanent exclusion. Lab, heart, kidney, liver, antibody, vaccination and medication criteria may apply even when they are not prominent in recruitment advertising.
Ask for formal prescreening by the site. Do not coach performance on a functional test or hide treatment history; inaccurate screening can create safety risks and invalidate participation.
A practical checklist
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Read both inclusion and exclusion criteria in the registry and consent materials.
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Confirm the date on which age and other thresholds are measured.
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Provide the exact mutation report and complete treatment/trial history.
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Ask which screening tests can be repeated and which cannot.
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Delay travel plans until the site confirms a realistic prescreening match.
Questions to bring with you
Use these at the clinic, school meeting, equipment evaluation, program interview or benefits call. Write down the answers and who owns the next step.
- Which criteria can be checked from records before an in-person visit?
- How is ambulatory status defined in this protocol?
- Is a stable-dose or washout period required?
- Could prior gene therapy or antibodies exclude only this study or an entire platform?
- Who makes the final eligibility decision?
Important context
Individual needs, eligibility and safety can differ. Confirm the plan with the relevant Duchenne-experienced clinician, therapist, school team or program before acting.
Sources used for this guide
Direct links are included so families can check the original guidance and bring it to qualified professionals.
Content review: July 18, 2026. Medical labels, trials, benefits and programs can change after publication.
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