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Clinical Trials

Families and individuals living with Duchenne often feel a lot of hope about clinical trials, especially for a treatment that has the potential to improve function, prognosis, and quality of life. We are fortunate that now is a time where our hopes are increasing. But along with hope, families may experience an emotional rollercoaster that brings elation, anxiety, and frustration as we learn to navigate the clinical trial process.  

Maintain hope—we have made so much progress!—but try to have realistic expectations of clinical trials. Keep a focus on living each day well, as we buy time for people with Duchenne.


  • Not all participants will benefit from a clinical trial, and in some cases, there will be significant side effects.  
  • The goal of a clinical trial is not to treat any one person—instead, the goal is to gather data about the safety, dosing, and/or effect of a treatment in the entire group. 
  • In some studies, not everyone receives the potential therapy—this is called a placebo control study, and no one (not even the medical staff) know if any one person is getting the potential therapy or a placebo, which is something that looks and seems like the therapy but has no effect (such as a sugar pill).
  • In most studies, after the trial is over, there is no guarantee that the people in the study will get to keep taking the therapy.
  • A family’s best approach is education about the research goals, eligibility requirements, study process, and the pros and cons of participation. Such information comes from resources like PPMD and DuchenneConnect. But the primary source of information is open communication with the study team.
  • Not every patient who seems appropriate for a clinical trial will be chosen for that trial. Before a candidate can be enrolled in a clinical trial, there is a complex and rigorous screening process. There are no guarantees that a candidate will be enrolled until the screening data has been evaluated.

This section provides you with basic information about the purpose of clinical treatment trials and why they are crucial for new treatment development. Study enrollment is explained, and an overview of the clinical trial process is provided. For more detailed information about clinical trials, including the risks and benefits of participation, we encourage you to visit the DuchenneConnect site.

In this section

Clinical Trial Process
How We Know When Something Works

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