Parent Project Muscular Dystrophy and Sarepta Therapeutics Join Forces on
Genetic Testing Program for Patients With Duchenne Muscular
HACKENSACK, NJ and CAMBRIDGE, MA -- (Marketwired) -- 10/17/13 --
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:
-- 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)
-- 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
-- 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.
PPMD Media Contact:
Sarepta Media Contact:
Sarepta Investor Contact:
Press spacebar to pause and continue. Press esc to stop.