Enter family, medical, and functional details to preview how DMD Pathways could surface trial options, explain why a family may or may not match, and make next steps easier to understand.
Trial C – ADVANCE Steroid Optimization & Cardiac Monitoring Study
Boston, USA + hybrid option • Longitudinal supportive-care and real-world evidence study
Trial A – NorthStar Microdystrophin Gene Transfer Study
London, UK • One-time interventional gene transfer study
Trial B – STRIDE Exon Skipping Therapy Study
Paris, France • Repeated dosing exon-skipping therapy study
DMD Pathways Navigation Support
Personalized trial-readiness checklist, qualifier explanations, records needed for outreach, and plain-language visit burden preview.
Travel & Reimbursement Planning
Questions to ask sites: prepaid vs reimbursed travel, lodging caps, caregiver meal coverage, accessible transport, sibling care, and timing of payment.
Genetic report, steroid history, cardiac testing, pulmonary function, 6MWT/NSAA, medication list, prior therapy exposure, and recent labs.
London, UK • One-time interventional gene transfer study
AmbulatoryAges 6–11Gene transferPossible overnight stayThis trial may focus on ambulatory boys/young men within a defined age range, with confirmed DMD, sufficient walking function, stable steroids, no prior gene therapy, and acceptable safety labs/cardiac review.
Window: Before screening • Length: 30–60 minutes by phone/video
Overnight guidance: No overnight expected.
Window: Day -60 to Day -30 • Length: Full clinic day
Overnight guidance: Possible if traveling long distance or if morning testing is required next day.
Window: Day -30 to Day -7 • Length: Half to full clinic day
Overnight guidance: Usually no, but may be needed if repeat cardiac/lab testing is scheduled.
Window: Day 1 • Length: Long day; may include inpatient or observation period
Overnight guidance: Overnight admission or nearby lodging may be required for safety monitoring.
Window: 7 days after dosing • Length: Half to full clinic day or hybrid
Overnight guidance: Usually no unless travel is difficult or multiple assessments are grouped.
Window: 30 days after dosing • Length: Half to full clinic day
Overnight guidance: Possible for long-distance families.
Window: Quarterly through Year 1 • Length: Half to full clinic day
Overnight guidance: Possible for combined assessments or long travel.
Window: Years 2–5 or longer depending protocol • Length: Often annual; remote/hybrid may be available
Overnight guidance: Usually not unless major assessments are bundled at site.
Travel may be arranged by a study travel vendor. Lodging for the patient and one caregiver may be covered for screening, dosing, and key follow-up visits. Meals, parking, mileage, and accessible transport may be reimbursed with receipts. Some visits may require overnight lodging, especially dosing and early safety follow-up.
| Family question | Why it matters |
|---|---|
| Is travel prepaid or reimbursed after receipts? | Families may not be able to front airfare, lodging, accessible transport, or hotel costs. |
| Are caregiver meals, parking, mileage, and lodging covered? | Small uncovered costs accumulate quickly over repeated visits. |
| Are remote, local lab, or home-health options available? | Hybrid design can reduce missed work, school disruption, and travel burden. |
| Is there a stipend for patient time and reporting? | Follow-up engagement depends on recognizing the real burden of participation. |
Timing/window: Day -60 to Day -30
Estimated time at site: Full clinic day
Possible overnight visit guidance: Possible if traveling long distance or if morning testing is required next day.
No IP dispensed.
Travel/lodging may be covered; receipts may be required.
This prototype flow shows how a family could ask DMD Pathways to forward their contact details and relevant intake information to the trial sponsor, biotech/pharma company, or site team.
Plain-English consent summary: By checking the box below, you are asking DMD Pathways to send your contact information and selected intake details to the trial sponsor, biotechnology/pharmaceutical company, contract research organization, or clinical trial site connected with the trial you selected so they may contact you with more information.
Important legal disclaimer: DMD Pathways is an informational navigation resource only. DMD Pathways does not provide medical advice, does not recommend or endorse a specific clinical trial or treatment, does not guarantee eligibility, access, enrollment, reimbursement, safety, or outcomes, and does not replace advice from your physician or the clinical trial site. The receiving sponsor/site is responsible for determining whether you may proceed to screening and whether additional consent forms, privacy notices, medical releases, or authorizations are required.
You may withdraw this request at any time by contacting DMD Pathways. Submission of this request does not enroll you in a study and does not create a patient-provider relationship with DMD Pathways.
Clicking medical terms throughout the prototype brings families here or opens a plain-English explanation.
The 6-minute walk test measures how far someone can walk in six minutes. It is commonly used in ambulatory Duchenne trials to measure function over time.
An echocardiogram is an ultrasound picture of the heart. It helps doctors see how well the heart is pumping and whether heart function is stable enough for a study.
FVC means forced vital capacity. It is measured during pulmonary function testing and helps show how much air a person can forcefully breathe out.
An infusion means medicine is given through an IV into a vein. Some infusions take several hours and require monitoring before and after.
Investigational product means the study drug, gene therapy, placebo, or other product being tested in the clinical trial.
Labs usually mean blood or urine tests. They may check liver, kidney, muscle, immune, or safety markers.
LVEF means left ventricular ejection fraction. It is a number that estimates how much blood the main pumping chamber of the heart pushes out with each beat.
MRI stands for magnetic resonance imaging. It is a scan that uses magnets and radio waves to create detailed pictures inside the body. In Duchenne studies, it may be used to look at muscles, the heart, or other organs. It does not use radiation, but it can be loud and may require lying still for a long time.
A muscle biopsy is a procedure where a small sample of muscle tissue is removed and tested. Some studies use it to look for dystrophin or other markers.
NSAA stands for North Star Ambulatory Assessment. It is a set of movement tasks used to understand walking, climbing, standing, and other functional abilities in ambulatory Duchenne.
An overnight visit means the patient may need to stay at or near the site overnight, often for safety monitoring, early morning testing, infusion observation, or multi-day assessments.
A PRO is information reported by the patient or family about symptoms, daily function, quality of life, or treatment burden.
A placebo looks like the study treatment but does not contain the active study medicine. Placebos are sometimes used so researchers can compare outcomes fairly.
Reimbursement means families may be paid back for approved costs such as travel, lodging, meals, mileage, parking, or childcare. Some studies reimburse after receipts, while others may prepay travel.
Screening is the step where the site checks medical records, tests, labs, and trial rules to see whether someone can enroll.
A washout period is a required waiting time after stopping another therapy or trial before starting a new study.
An overnight visit means the patient may need to stay at or near the site overnight, often for safety monitoring, early morning testing, infusion observation, or multi-day assessments.
This optional feedback helps show where families disengage and what barriers need to be solved earlier.