Details Regarding Study DMD114117 for Duchenne Muscular Dystrophy

Details regarding several of the investigator sites for the following study have been posted to www.orpha.net (the portal for rare diseases and orphan drugs) today:  

Study DMD114117: A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy

Additional study details, including inclusion/exclusion criteria and study endpoints are posted on www.clinicaltrials.gov.  Investigator site details for this study, taking place in Europe, Turkey and Australia, will also appear on www.clinicaltrials.gov in the near future.

Sincerely,

 John E. Kraus, MD, PhD                                Padraig Wright, MD, PhD

john.e.kraus@gsk.com                                 padraig.x.wright@gsk.com

New Cure Duchenne Research Index

CURE DUCHENNE ANNOUNCES FREE ACCESS TO THE CURE DUCHENNE RESEARCH INDEX

CureDuchenne completes Duchenne Muscular Dystrophy Advisory Board with stem cell specialist and simultaneously announces the release of a comprehensive DMD research index

 Click here to see the new Research Index 
(also found under the Research Heading)

CureDuchenne, a nonprofit dedicated to moving the needle on Duchenne Muscular Dystrophy (DMD) research in order to create real change for this generation of boys affected by the disease, today announced the completion and release of the CureDuchenne Index, the DMD community’s first end-to-end index documenting all DMD research currently taking place around the globe.

DMD is the leading genetic killer of children and affects 1 in 3,500 boys. DMD is usually diagnosed by the age of five, the boys are often relegated to a wheelchair by age 12, and may be completely paralyzed by their late teens. Historically, most patients with DMD do not live to see adulthood. CureDuchenne’s mission of funding research projects on the cusp of becoming human clinical trials in an effort to create real change in treatment options now, has established them a leader in the scientific fight against DMD.

The public, DMD organizations and research communities may link to the CureDuchenne Research Index:   http://www.cureduchenne.org/research/research-index/.    The CureDuchenne Research Index is intended to serve as a resource for parents, advocates, scientists and fundraisers as they work together toward a cure for DMD.

“So much important work is taking place around the world and we felt there wasn’t one place to go where you could capture it all,” said CureDuchenne co-founder Debra Miller. “Working collaboratively is the only way we’ll make a dent in this disease and I wanted CureDuchenne to extend that hand. This index has been invaluable to us and I hope that others will also benefit from being able to see the big picture around DMD research.”

The completion of the CureDuchenne Index aligns with the selection of an additional scientist for CureDuchenne’s world-class scientific advisory board. Designed with each segment of DMD research in mind, CureDuchenne has augmented their board with the addition of stem cell research expert Thomas A. Rando, M.D., PhD of Stanford University.  Rando, a professor in the Department of Neurology and Neurological Sciences at Stanford University School of Medicine, Chief of the Neurology Service at the VA Palo Alto Health Care System, Deputy Director of the Stanford Center on Longevity (SCL) and graduate of Harvard Medical School, has worked extensively with stem cells to create muscle regeneration and growth, a key factor in the fight against DMD.

“Joining the CureDuchenne advisory board is synergistic with the work I’ve been doing with muscle growth and regeneration,” said Rando. “The boys that face DMD have a big challenge in front of them and as scientists we want to work as quickly as possible toward a cure. CureDuchenne is the kind of organization that identifies projects that have a real chance of being successful and as a result, they’ve helped our field make great strides. I’m honored to be included among the top-notch scientists on their advisory board.”

CureDuchenne’s scientific advisory board works with the organization to determine what projects around the world are closest to the human clinical trial phase and the most viable for funding.  To date, CureDuchenne has provided millions of dollars in funding for DMD research around the world and has been integral in bringing 3 treatment options to the human clinical trial phase.

The CureDuchenne advisory board will meet next at the 2^nd Annual DMD Summit in Orange County, California. The three-day Summit, set to take place January 27-29, 2011, will include closed session discussions, research project evaluation and a breakfast event where each participant will report on the findings from the weekend. The breakfast will be attended by DMD parents, advocates and supporters.

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GSK Anticipates Delay in 48 week Phase 3 DMD Study

ON BEHALF OF JOHN KRAUS, MD, PHD and PADRAIG WRIGHT, MD, PHD

(GLAXOSMITHKLINE)

Today, GSK informed a number of potential study investigators that we anticipate a delay in the planned 48 week phase 3 study (Study DMD114044) of GSK2402968, commencing in Canada. 

Health Canada has requested that GSK provides additional data to inform their decision regarding approval of initiation of Study DMD114044.  This study is planned to investigate the safety and efficacy of GSK2402968 in ambulant boys with DMD, who have a dystrophin gene mutation amenable to an exon 51 skip. 

GSK is working with Health Canada to provide the data they require to initiate the study in Canada as soon as possible. 

In the meantime, GSK is working with other regulators and progressing with plans to begin the study outside of North America.  GSK will alert you when study details, including study site locations, are posted to www.clinicaltrials.gov. 

 John E. Kraus, MD, PhD                                  Padraig Wright, MD, PhD                                          
john.e.kraus@gsk.com                                    padraig.x.wright@gsk.com

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