Do you or a loved one have a diagnosis of Becker muscular dystrophy? If so, please read these important updates which are focused on how we can help individuals with Becker and their families.

Becker Research Update

There is much excitement in the news lately about the robust drug development pipeline for Duchenne, but there are also several clinical trials and research studies that are actively recruiting or soon to be recruiting individuals with Becker.

A new study that is actively recruiting adult males (ages 18-62 years old) with Becker is MRI and Biomarkers for Muscular Dystrophy. This study focuses on developing Magnetic Resonance Imaging (MRI) and spectroscopy (MRS) as a tool to monitor disease progression in muscular dystrophy and to serve as an outcome measure for clinical trials. This study is recruiting at two different sites in the US: Gainesville, FL at the University of Florida (UF) and Portland, OR at the Oregon Health and Science University (OHSU). This study is funded by the National Institutes of Health – NIAMS/NINDS. A total of 105 adult men with Becker are needed, and participants will be paid a total of $500 over the course of 3 visits, as well as your travel and accommodations.

Below are some of the investigational products being evaluated for individuals with Becker:

• Givinostat (Italfarmaco) – Givinostat has been evaluated in a Phase 2 study in Becker patients in Europe. Italfarmaco shared an update at the 2021 PPMD Annual Conference on the initial results and reporting that they will be working with regulatory authorities to discuss development plans for the use of Givinostat in adults with Becker.
• EPM-01 (Epirium Bio) – EPM-01 is currently being studied in a fully recruited Phase I trial evaluating safety and early efficacy in patients with Becker.
• EDG-5506 (Edgewise Therapeutics) – Edgewise is actively recruiting both adult healthy volunteers and adult individuals with Becker for a Phase I trial evaluating the safety of EDG-5506.
• Vamorolone (ReveraGen/Santhera) – Recently, ReveraGen and Santhera shared news of their FDA grant to support a clinical trial to evaluate the use of Vamorolone in patients with Becker. The placebo controlled study will take place at the University of Pittsburgh, USA and University of Padova, Italy.

Join The Duchenne Registry

A great way to help advance research and stay informed about clinical trials is by joining The Duchenne Registry. The Registry is not only for individuals with Duchenne, but also for those with Becker and carrier females. We have approximately 400 individuals with Becker in our Registry, but we have over 4,000 individuals with Duchenne. We need more Becker data in the Registry! The Registry data is utilized by researchers around the world to help improve care and advance research and treatments for Duchenne and Becker, so please consider joining today. All data entry occurs through our easy-to-use app! Learn more on our website and start the registration process by clicking Join. If you have any questions, please contact the Registry team at 888-520-8675 or coordinator@duchenneregistry.org.

Watch our Registry video to learn more about why your data is important!

Get Social!

Did you know that we currently have three men on our PPMD Adult Advisory Committee (PAAC) with Becker? We have a Facebook group for adult men in the Duchenne and Becker community, and a new social networking platform called PAAC Chats to help you stay connected with other adult men in the Duchenne and Becker community. If you are a teen or adult with Duchenne or Becker age 16 or older, download PAAC Chats today in the apple or google play stores, or participate via web.

At PPMD, we are continually brainstorming how to benefit and engage the Becker community. If you have any ideas to share, please reach out to Pat Moeschen at pat@parentprojectmd.org.