NEW Duchenne Muscular Dystrophy
are enrolling now!
Clinical trials are often viewed as the best treatment option for a muscular dystrophy patient, so it is important you learn about them as a treatment option to help make an informed treatment decision. Deciding to participate in a clinical trial is a big step. It’s an important choice for you and your child – and it supports the advancement of medical science and the improvement of healthcare for everyone.
Don’t Waste Your Time
Your loved one may qualify IF
  • He or she is 5 years of age or older
  • Was diagnosed with Duchenne Muscular Dystrophy (DMD)
  • You’re ready to try an investigational treatment for Duchenne Muscular Dystrophy (DMD)
Benefits to you if you qualify
100% FREE
You’ll receive study-related medical care and medication at no cost. It is 100% free; no insurance is needed.
You’ll be monitored by doctors who are experienced in treating Duchenne Muscular Dystrophy (DMD).
Your only investment is time, and you are always free to opt out of the study with no further obligation.
‘‘No child deserves this.’’
Jessica A.
Note: All information provided will be kept confidential, except when ordered by law, and only accessible to our clinical trial partners, and applicable regulatory agencies.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. It is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disease primarily affects boys, but in rare cases it can affect girls.
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DMD affects about one in 5,000 males at birth, It is the most common type of muscular dystrophy.

Researchers are actively pursuing several exciting strategies in DMD, such as gene therapy, exon skipping, stop codon read-through and gene repair. Human clinical trials are under way for some of these strategies.

We may be able to help
find the right trial for you
Give a DMD clinical trial a chance.
It is 100% free.
About the Studies

The Studies are evaluating an investigational medication of Duchenne Muscular Dystrophy (DMD). These studies will help determine if the investigational medication is safe and effective at curing or slowing the progression of the condition.

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A clinical trial is carefully designed to closely monitor patients’ progress as they go through treatment with an investigational drug, product, device or method of treatment that has not been approved by the FDA. Clinical trials help find new ways to treat, prevent and diagnose diseases. Today, all medications prescribed by a doctor must first be tested in clinical trials. Study participants receive close medical supervision and provide valuable feedback on their experiences.

All clinical trials in the United States are reviewed by the FDA and other governing bodies called Institutional Review Boards, whose job is to make sure participants’ rights are fully protected and that participants are not exposed to any unnecessary risks.

All treatments must go through three phases of clinical research before becoming available to the public:

Phase I
focuses primarily on safety in a small number of human volunteers
Phase II
tests the effectiveness of the new drug on a small number of human volunteers
Phase III
usually tests the new drug in comparison with the standard therapy currently being used on a larger number of human volunteers.
Please note there are Phase III studies currently enrolling in the USA
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Participating in a clinical trial is voluntary. Participants may choose to discontinue participation at any time. Before you enroll in a trial, a doctor or nurse will clearly explain the study procedures and requirements. This is called informed consent. Doctors are not allowed to enroll patients in clinical trials without first ensuring they understand what their involvement in the trial means. Once you enroll, you may end your participation at any time.

When participating in a clinical trial, participants receive study-related medications and medical monitoring at no cost. Most studies reimburse for travel and logistical considerations. It is important to find out the insurance requirements of any study you want to participate in.

‘‘Nothing on earth is harder for a parent than watching their child dwindle away’’
Andrea V.
Why Participate?

There are a number of reasons you or your doctor may want to consider participation in a clinical trial, including:

  • Access to investigational treatments before they become widely available in most cases at no cost for patients. No insurance is usually needed.
  • Opportunity to play a vital role in the discovery of treatments, cures and preventions of diseases or medical conditions.
  • Ability to play a more active role in your own healthcare.
  • Access to free physical examinations and diagnostic tests related to the study.
  • According to a survey by the Center for Information & Study on Clinical Research Participation (CISCRP), 95% of study volunteers would consider participating in future clinical trials.
Is your child
diagnosed DMD?
Please fill out the Enroll form and let
us check if there is a clinical trial
available nearby.
how it works?
Sign up for a study.
Submit a short profile by providing basic info about your child and your brief medical history.
See if you pre-qualify.
We will check the clinical trials you pre-qualified or notify you when one becomes available.
Contact you to participate.
We will match you to a clinical trial coordinator in your location and you will then have the opportunity to participate.
Note: All information provided will be kept confidential, except when ordered by law, and only accessible to our clinical trial partners, and applicable regulatory agencies.
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    Does the participant have to pay to participate in the Duchenne Muscular Dystrophy (DMD) study?

    No. The participant will receive all study-related care, including the investigational medication, at no cost. Insurance is not needed to participate in the study and the participant’s insurance company will not be notified.

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    Will the participant be paid for taking part in the study or reimbursed for travel?

    Reimbursement for travel expenses may be provided. If pre-qualified, the study team will provide more information.

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    Will the participant’s doctor have to give permission to participate?

    No. The participant’s doctor does not have to give his or her permission to take part in the study. However, either the participant or the study doctor, with the participant’s permission, may contact the personal doctor to discuss the participant’s participation and progress throughout the study.

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    What is an investigational medication?

    An investigational medication is a medication that has not been approved by the appropriate regulatory or health authorities, such as the U.S. Food and Drug Administration (FDA), for prescription use. Investigational medications are yet to be proven safe and effective.

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    Am I going to get a sugar pill (placebo) while other patients are getting the real medication?

    Studies will only use placebos when absolutely necessary. In 99% of clinical trials, if a placebo is used, it is given with standard treatment. You will receive either the best known Duchenne Muscular Dystrophy (DMD) treatment or a new and possibly more effective treatment. Many trials even combine the standard therapy with a new treatment.

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    Are clinical trials too risky for me?

    All procedures that address a life-threatening illness pose some risk. Your physician and Duchenne Muscular Dystrophy (DMD) treatment team might help you decide if a clinical trial is the best treatment option for you. In muscular dystrophy diagnosis, clinical trials are often viewed as the best treatment option. Keep in mind that all known medications have once gone through the clinical trials process - even aspirin.

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    Can I suspend my participation in the study at any time?

    Yes, participation is voluntary. The participant may decide to stop or suspend participating at any time. Any participant who decides to stop early should give prior notice to the study team. The study team will ask the participant to return to the study office at least once for a follow-up test to check the participant’s health once the investigational medication has been stopped.

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    If I participate in a Duchenne Muscular Dystrophy (DMD) clinical trial, my child may be treated like a guinea pig.

    Not at all! Participating in a clinical trial is always voluntary. You will go through a detailed informed consent process and you may always choose to stop participating. Most people like being on a clinical trial because they feel they are being monitored even more closely than before.

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    Who oversees the Duchenne Muscular Dystrophy (DMD) clinical trials?

    To help ensure the clinical trial is ethical and that participants’ rights are protected, Institutional Review Boards (IRB) or Ethics Committees (EC) review and approve study protocols.

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    What happens to our personal information?

    The study team respects and protects every participant’s privacy. The study team will not share the participant’s information except when they are required by law. The study team will store the participant’s personal information with codes that do not identify the participant. The Informed Consent Form has more information about the participant’s privacy and you will need to sign it prior being accepted into the study.