Decode Duchenne Announces 1,000th Patient Application

HACKENSACK, N.J., June 25, 2018 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced the 1,000^th patient application to their highly successful Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with Duchenne muscular dystrophy in accessing genetic testing, interpretation, and counseling.  The program was founded in partnership with Sarepta Therapeutics in 2013 and has continued to expand with continued support from Sarepta Therapeutics and the help of PTC Therapeutics.

Decode Duchenne provides genetic testing, interpretation, and counseling at no cost to eligible patients who are unable to access testing due to barriers such as a lack of insurance or insufficient insurance coverage. For people with Duchenne, genetic testing is used to identify the genetic mutation and confirm the diagnosis. In addition, genetic testing can be used not only to determine a person's eligibility for certain clinical trials, but also their compatibility for potential therapies currently available or on the horizon. Despite the benefits of testing, in some cases patients are unable to access genetic testing through their insurance provider.

Until 2018, the Decode Duchenne program was only available to those with a confirmed or suspected diagnosis of Duchenne or Becker muscular dystrophy. A new addition to the program in 2018 is carrier testing for at-risk females who have a relative affected with Duchenne. Women often have trouble accessing carrier testing, which is critical for accurate family planning.  Including carrier testing in the Decode Duchenne program enables PPMD to provide genetic testing, interpretation, and counseling for the entire family affected by Duchenne. Applicants typically must also have financial barriers to receiving genetic testing and be citizens or legal residents of the United States or Canada.

PPMD's Founding President and CEO, Pat Furlong, is moved by this milestone for Decode Duchenne: "This is a meaningful moment for this important community program. To have reached 1,000 patients speaks to the incredible need of free genetic testing and counseling in our community, as well as the talent and compassion of the Decode Duchenne team. Since the initial start-up phase of Decode Duchenne in 2013, we have been able to identify people with Duchenne within populations that traditionally have not had access to genetic testing, interpretation, and counseling because of costs associated with the process. With the initial and ongoing support of Sarepta Therapeutics, and the partnership of PTC Therapeutics in 2015, we have seen incredible growth in the program."

The program is administered by PPMD's board-certified genetic counselors Jennifer Ely and Ann Martin. Ms. Martin, director of The Duchenne Registry believes that the Decode Duchenne program is more important than ever: "More potential therapies are emerging every day for Duchenne and many of these are based on your individual genetic change. Knowing your genetic mutation gives you the knowledge to move forward as new therapies emerge and possibly find one that is right for you. The steady increase in applications and call volume highlight the importance of the program in our community.  The Decode Duchenne team and sponsors will continue to find ways to expand our mission to improve diagnostic efficiency and accuracy at all ages and stages."

To learn more about Decode Duchenne, including how you or a loved one can participate, please visit ParentProjectMD.org.

About Decode Duchenne

PPMD's Decode Duchenne genetic testing program provides free genetic testing and counseling to people in the Duchenne or Becker muscular dystrophy community who otherwise could not afford genetic testing. Our process is simple: have your local provider complete our application and receive all the information and paperwork needed to have genetic testing performed through our designated laboratory, PerkinElmer Genetics.

Our genetic testing includes full analysis of the Duchenne gene through next generation sequencing, targeted testing, or repeat testing for those with incomplete testing thus far. Our program can also provide specimen shipping kits and reimburse blood draw costs when needed. Two certified genetic counselors are also available to providers and families at any point during the testing process.

Decode Duchenne is administered by Parent Project Muscular Dystrophy and The Duchenne Registry. Decode Duchenne is sponsored by Sarepta Therapeutics and PTC Therapeutics.

To learn more about Decode Duchenne, including information on how to participate, visit ParentProjectMD.org.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.

About The Duchenne Registry

The Duchenne Registry (formerly DuchenneConnect) began in 2007, when a group of thought leaders in the Duchenne and Becker muscular dystrophy community began discussing the need for a new kind of resource that would connect and serve the needs of the entire community. What they envisioned was a central hub that would bring together those living with Duchenne or Becker, along with their families and caregivers, to connect them with medical research, clinical care, clinical trials, and each other. At the same time, it would also be a resource for researchers and industries with an interest in Duchenne, allowing access to aggregate, de-identified data provided by patients and their families — information that could prove vital to advances in care and treatment. Today, the result of this endeavor is The Duchenne Registry, the largest, most comprehensive registry for Duchenne and Becker muscular dystrophy, as well as female carriers.

Ann Martin, Director of The Duchenne Registry, adds that the success of this platform is a direct result of community participation: "When families join and update their account in The Duchenne Registry, they are strengthening the power of a 10-year-old network of patient-powered data that will be used to improve care for people living with Duchenne and increase understanding of the disorder. Families become citizen scientists by contributing to real scientific research, without ever leaving their homes."

To learn more about The Duchenne Registry, visit our website.

SOURCE Parent Project Muscular Dystrophy

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