Parent Project Muscular Dystrophy and Sarepta Therapeutics Join Forces on Genetic Testing Program for Patients With …

Posted: Published on October 17th, 2013

This post was added by Dr Simmons

HACKENSACK, NJ and CAMBRIDGE, MA--(Marketwired - Oct 17, 2013) - Parent Project Muscular Dystrophy (PPMD) and Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today announced a new nationwide program from PPMD to assist individuals with Duchenne muscular dystrophy in accessing genetic testing. The program will be administered by PPMD, a leading voice for patients and families impacted by Duchenne. Support for the initiative will be provided by Sarepta, a developer of innovative RNA-based therapeutics.

Through the new program, called Decode Duchenne, PPMD will offer genetic testing at no cost to eligible patients who are unable to access testing due to barriers such as a lack of or insufficient insurance coverage. Decode Duchenne is expected to launch in the fourth quarter of 2013 through PPMD's DuchenneConnect, an online resource and registry for patients and families affected by Duchenne and their healthcare providers. For patients with Duchenne, genetic testing is used to identify the genetic mutation and confirm the diagnosis. In addition, genetic testing can be used to determine a patient's eligibility for certain clinical trials. Despite the potential benefits of testing, in some cases patients are unable to access genetic testing through their insurance provider.

"We believe all patients with Duchenne should have the opportunity to receive genetic testing in order to understand their genetic mutation, have confidence in their diagnosis and learn about opportunities to participate in clinical trials of promising investigational treatments," said Pat Furlong, president and chief executive officer of PPMD. "This program seeks to address barriers to genetic testing that have long frustrated the Duchenne community and to help vulnerable patients and families."

"We are pleased to support Decode Duchenne, an important initiative from PPMD and DuchenneConnect to help families and their physicians navigate a sometimes difficult process to receive genetic testing," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "Sarepta's support for this new program is part of our broader commitment to develop new potential therapies, as well as provide education and resources, that may help to improve outcomes for boys and young men with Duchenne."

To participate in Decode Duchenne, patients must:

Additional eligibility criteria may apply, and the DuchenneConnect Coordinator can help interested patients and their families determine if they are eligible to participate.

Patients and families can learn more about the Decode Duchenne genetic testing program by visiting DuchenneConnect at http://www.duchenneconnect.org, or by contacting the DuchenneConnect Coordinator at coordinator@duchenneconnect.org or (201) 937-1408. Information is also available through Sarepta's new online resource center Let's Skip Ahead, which is available at http://www.skipahead.com.

About DuchenneConnect

DuchenneConnect is a robust and cutting-edge registry and resource that serves the needs of the Duchenne and Becker community. The purpose of DuchenneConnect is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, DuchenneConnect is a valuable resource for clinicians and researchers in academia and industry, allowing access to aggregated, de-identified information provided by patients and their families -- information that is vital to advances in the care and treatment of Duchenne.

DuchenneConnect was created in 2007 by Parent Project Muscular Dystrophy (PPMD), with assistance from the Centers for Disease Control and Prevention and Emory Genetics. PPMD is the sole guardian of DuchenneConnect and its material. To learn more about DuchenneConnect, visit http://www.duchenneconnect.org.

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