The role and importance of natural history studies in clinical development

We invite you to watch this video to learn more about the role of natural history studies in drug development and a family's decision to participate in JOURNEY, Sarepta's natural history for LGMD types 2E/R4, 2D/R3, 2C/R5, and 2A/R1. If you are interested in learning more, you may email Advocacy@Sarepta.com and visit the study listing on clinicaltrials.gov.

What is a natural history study?

A natural history study does not involve the use of an investigational study drug and instead studies the natural progression of a disease over a period of time. JOURNEY is a natural history study that Sarepta is conducting to better understand the natural progression of limb-girdle muscular dystrophy types 2E/R4, 2D/R3, 2C/R5, and 2A/R1. The data collected from JOURNEY will help Sarepta learn more about the condition and how muscle strength, breathing, and heart function change over time.

Why are natural history studies, such as JOURNEY, important?

Natural history studies help clinicians and researchers better understand a disease, and they can be especially valuable for rare diseases that are not already well characterized due to their low prevalence.

In addition, information collected from natural history studies plays an important role in drug development. For example, it could be difficult to determine how an investigational study drug has made a difference in disease/symptom progression without natural history data to compare to.

If you/your loved one is living in the United States (U.S.) and wish to sign up for updates about Sarepta’s work and community educational resources, please visit https://www.limbgirdle.com/stay-connected. For community members in the U.S. or living outside the U.S. wishing to speak to a member of Sarepta’s Patient Affairs team, please email Advocacy@Sarepta.com.