Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as well. We need more sensitive outcome measures that are more inclusive to a broad population of individuals living with Duchenne and Becker. We also need to be measuring more than muscle strength because we know how important things like quality of life also are for those living with Duchenne and their families.

During the “Outcome Measures: The ERAs Tour” session at PPMD’s 30th Annual Conference, experts discussed the latest advancements in outcome measures for Duchenne. The session highlighted the importance of accurate and reliable outcome measures in assessing the effectiveness of therapies and guiding clinical trials. Presenters explored various tools and methodologies used to track disease progression and patient response to treatments. Emphasis was placed on the need for standardized and patient-centered outcome measures to ensure meaningful data collection. This session provides valuable insights into how improved outcome measures can accelerate the development of effective treatments and enhance patient care.

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During the “Clinical Trials: A Panel Discussion on the Developmental Path” session at PPMD’s 30th Annual Conference, a panel of experts delved into the complexities of developing clinical trials for Duchenne. The discussion covered the various stages of clinical trial development, from initial design to implementation and data analysis. Panelists highlighted the challenges and opportunities in recruiting participants, ensuring patient safety, and meeting regulatory requirements. The importance of collaboration between researchers, clinicians, and the Duchenne community is emphasized as key to advancing successful trials. This session offers valuable insights into the critical role of clinical trials in bringing new therapies to patients.

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Clinical Trial Resources for You

PPMD’s Understanding Clinical Trials page offers helpful information about trial participation, including risks, benefits, and inclusion and exclusion criteria, as well as informed consent and a broad overview of what to expect during a clinical trial.

Understanding Clinical Trials

You can explore current clinical trials here. Additionally, the Genetics, Clinical Trials, & The Duchenne Registry appointments through our PPMD For You program provide an opportunity to meet one-on-one with PPMD’s genetic counselors for an overview of the current clinical trial landscape. We can work together with you to identify potential clinical trials that may be worth considering for your family.

PPMD For You

If you are considering a clinical trial for you or your child, PPMD’s Clinical Trial Decision Guide is a great tool to help you decide whether or not clinical trial participation is right for you and your family.

You and your family should know as much as possible about the research study or clinical trial and should feel comfortable asking your study team questions. It is important to ask the study team questions about the goals of the trial and the likelihood of any benefits during and after the trial. Find a list of questions for your meetings with your clinical trial team here.

Considering a Clinical Trial