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Research & Clinical Trials
There are many ways to help move Duchenne science forward — and not all of them involve taking an experimental medicine.
What Is Clinical Research?
Clinical research is the work scientists do to understand diseases and discover better treatments. It is how every current Duchenne therapy became available — and how tomorrow's therapies will be found.
Researchers need families.
Better treatments cannot be discovered without people who are willing to participate. Your family's involvement is valuable and necessary.
There are many ways to take part.
You can join a trial, enter a registry, complete surveys, share your story, or simply allow your child's health information to be counted.
It doesn't always mean a new medicine.
Some research studies observe, listen, or learn. Families are not always asked to take an experimental drug.
Clinical Trials
A clinical trial is a carefully designed research study that tests whether a new treatment is safe and whether it works. Trials are the final step before a new therapy can be approved and made available to families.
Why They Matter
- They are how new Duchenne therapies are proven safe and effective
- They can give families early access to promising treatments
- They produce the evidence doctors need to recommend new standards of care
- They move science forward for every child diagnosed in the future
The Phases
- Phase 1Safety. A small number of participants help researchers learn about side effects and dosing.
- Phase 2Early signals. Researchers look for signs that the treatment may work.
- Phase 3Larger proof. The treatment is compared against current care to confirm benefit.
Why some children qualify and others don't
Every trial has rules about age, mutation type, ambulatory status, medications, and where a family lives. Those rules exist to protect participants and keep the results clear. Not qualifying is normal — it is not a rejection of your child. Most families will not match every trial they read about.
Patient Registries
Almost every family can participate
A patient registry is a secure database that collects information about people living with a specific condition. It is one of the most important — and most overlooked — ways families can help Duchenne research.
What a registry is
A registry is a bank of health information — diagnosis, mutation, medications, function, and daily life — stored securely and shared with researchers who are working on Duchenne.
Why researchers need them
Scientists need to understand the full picture of Duchenne to design trials. Registries show them how the disease progresses, what families need, and where to find eligible participants.
How they identify eligible families
When a new trial opens, researchers can use registry data to find families who might match. That means a registry can bring opportunities directly to you instead of you searching for them.
How they accelerate research
A well-populated registry can shorten the time it takes to launch a trial, answer regulators' questions, and get new treatments to families faster.
Why keeping your registry information up to date matters
Your child changes over time, and so does research. A registry entry that is current can help scientists answer new questions, design better trials, and reach your family when the right opportunity opens up.
Natural History Studies
Sometimes researchers simply observe how Duchenne changes over time. There is no experimental drug. No randomization. Just careful learning.
What They Involve
- Regular clinic visits, measurements, and tests over time
- Tracking how movement, heart, lung, and bone health change
- Collecting samples or imaging that show the disease's progression
- No new treatment assigned by the study
Why They Matter
Natural history studies have been responsible for many of today's advances. They taught researchers what to measure in trials, how fast Duchenne progresses, and what endpoints truly matter to families. The knowledge from these studies is the foundation new treatments are built on.
Surveys & Patient Voice
Families contribute to research by sharing what daily life is really like. Your experience changes how trials are designed and what questions scientists ask.
Your voice shapes the science.
When enough families share their experiences, researchers and regulators learn what really matters. Surveys have changed how clinical trials are designed, what symptoms are measured, and how new treatments are approved.
Advocacy & Community Research
Research does not only happen in labs. It also happens when families speak up, advise organizations, and help shape the questions the community asks.
Community organizations
Patient advocacy groups collect insights, fund studies, and connect researchers with families. They often run the registries and surveys that drive progress.
Patient advisory boards
Some trials and programs include family advisors who review materials, give feedback, and make sure research is respectful and practical.
Focus groups & listening sessions
Researchers and companies sometimes ask families to talk about what daily life is like. These conversations shape trial design, devices, and services.
Registry advisory panels
Families can help decide what a registry should ask, how data should be shared, and how the community should be informed about findings.
What We Wish Someone Had Told Us
You do not have to decide today. Most families explore trials, registries, and research more than once across their child's journey. New opportunities open every year.
Saying "not right now" is a complete answer. Participation is one option among many — not a measure of whether you are doing enough as a parent.
Bring your neurologist into the conversation. Your child's neuromuscular specialist can help you weigh what a specific study or trial would mean for your family.
When you're ready
Explore Trial Matching
Our Trial Matching tool walks you through a short intake about your child's age, mutation, stage, and location — then shows the Duchenne clinical trials that fit, anywhere in the world, in plain language.
Start Trial MatchingBuilt for families
No medical jargon. No sales calls.
Plain-language matches
We translate every eligibility rule.
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Your information stays yours.