HACKENSACK, NJ and CAMBRIDGE, MA--(Marketwired - Oct 17, 2013) - Parent Project Muscular Dystrophy (PPMD) and Sarepta Therapeutics, Inc. (
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:
- Have a confirmed diagnosis or be suspected of having Duchenne or Becker muscular dystrophy based on clinical symptoms, as assessed by their treating physician, and have a positive creatine kinase (CK) test.
- Have not previously had genetic testing, or must require additional genetic testing to identify a causative mutation, as determined by the DuchenneConnect Coordinator, a board-certified genetic counselor.
- Provide documentation to confirm the patient's lack of insurance coverage, insufficient insurance coverage, or a denial of coverage for genetic testing.
- Be citizens or legal residents of the United States.
- Register on DuchenneConnect.
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 www.duchenneconnect.org, or by contacting the DuchenneConnect Coordinator at firstname.lastname@example.org or (201) 937-1408. Information is also available through Sarepta's new online resource center Let's Skip Ahead, which is available at www.skipahead.com.
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 www.duchenneconnect.org.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men 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 young men 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 boys with Duchenne live longer, stronger lives. We will not rest until every young man 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.
About Sarepta Therapeutics
Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world's most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at www.sarepta.com.