Study of Genetic Modifiers of Duchenne and Becker

Genetic Modifiers of Duchenne and Becker Muscular Dystrophy

Questions and Answers

What stage is this research?

This study is actively recruiting participants.

What is the goal or purpose of this study?

The Genetic Modifier Study is trying to figure out what genetic changes are causing some people with Duchenne or Becker to be more mildly affected and others to be more severely affected. In other words, what genes are modifying the person’s disease. Researchers will try to identify genes and gene variants that may modify the disease process and that will move the community closer to find effective treatments for Duchenne and Becker.

Who is funding this study?

This study is funded by the NIH and PPMD.

Who is eligible to participate in this study?

All males (any age) with Duchenne or Becker are eligible to participate.

What do I have to do if I decide to participate in this study?

Participation in this study requires:

  • A brief one page questionnaire that is emailed to you, and returned by email.
  • Blood draw or saliva collected near your home, at UCLA or at your local doctors office.
  • A brief annual health survey (optional but helpful) by email or phone.

Where does this study take place?

This study takes place at the Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA in Los Angeles, CA.

Can any visits be done locally?

Yes. Study participants do not travel to UCLA. Participants complete all study procedures where they live.

Can I request a genetic report or my sequencing data to be sent to me?

No. This is a research study, no report will be generated and our protocol does not allow us to return their data to the participants.

On the other hand, if we find something clinically relevant we will discuss the findings with the participants.

Will I get paid for participating in this study?


Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a study, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), and helping others by contributing to the better understanding of Duchenne and Becker.

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