PTC Therapeutics and Genzyme Corporation Announce Preliminary Results from the Phase 2b Clinical Trial

March 3, 2010 by CureDuchenne
Filed under Research Articles

CureDuchenne’s Scientific Board of Advisors

February 27, 2010 by debramiller
Filed under Research Articles

Six years ago when Paul and I founded CureDuchenne, we committed ourselves to aggressively funding Duchenne muscular dystrophy research. We were well aware that not all of our research projects were going to be home runs, but part of science is taking that risk (not safety risks, of course) and getting as many strong research candidates funded as possible. We were prepared to look upon project failures as a positive aspect of keeping the pipeline full.

We are very pleased to say that all of our major funding projects are on course and moving forward. Three of our projects are in phase ll and phase lll clinical trials, with two more of our projects expected to reach trial in 2010. We have been very happy to collaborate with other organizations such as Charley’s Fund, FED, Jett Foundation and PPMD when appropriate, but there are times when waiting for a group decision would have slowed the process down. In the case of Prosensa, our ability to move quickly and independently sped up the development of exon skipping.

We owe a great deal of gratitude to our scientific advisory board for steering the CureDuchenne research ship toward impactful research projects. Our board is made up of independent advisors, all with expertise in DMD or drug development. We believe that drawing upon the expertise of a pool of experts with a diverse background and areas of specialization provides our organization with the most comprehensive and unbiased recommendations available.

On March 19, all of our advisors will meet in Newport Beach to review potential research and guide CureDuchenne on our funding priorities for 2010 and beyond. We will hold an informal breakfast meeting on Saturday, March 20 which will be open to the public to come and meet the scientists and hear the latest updates on Duchenne research. This will be an adult only meeting. Please click this link to register: http://www.breakfastwithdmdexperts.eventbrite.com/

I’m sure I speak for all parents of Duchenne boys when I say thank you to these scientists.

Debra

CureDuchenne’s Scientific Advisory Board

Barry J. Byrne, MD, PhD, University of Florida Professor and Associate Chair of Research,Department of Pediatrics and Microbiology & Molecular Genetics.
Kevin Campbell, PhD, University of Iowa Roy J. Carver Biomedical Research Chair in Molecular Physiology and Biophysics
Jeffrey Chamberlain, PhD, University of Washington School of MedicinePrinciple Investigator of the Chamberlain Lab in the Dept. of Neurology
Eric Hoffman, PhD, Chairman, Department of Integrative Systems Biology, George Washington University School of Medicine, Clark Chair and Director, Research Center for Genetic Medicine,Children’s National Medical Center in Washington DC
Edward Kaye, M.D. Group VP Clinical Research at Genzyme
Douglas Macdonald, PhD Director Drug Discovery at CHDI Foundation, Inc.
Carrie Miceli, PhD, UCLA Professor in the Dept. of Microbiology, Immunology & Molecular Genetics
Stanley Nelson, MD, UCLAProfessor in the Departments of Psychiatry & Biobehavioral Sciences & Human Genetics
Pier Lorenzo Puri, MD, PhD, Burnham Institute San Diego Ca. Assistant Professor at the Burnham Institute
Brian Tseng, MD, PhD, Massachusetts General Hospital Harvard Medical SchoolAssistant Professor & Director at Pediatric Neuromuscular Clinic