Prosensa and Professor Qi Lu to collaborate on ‘smart therapy’ for Duchenne Muscular Dystrophy
October 10, 2006
For immediate release
Leiden, The Netherlands, October 10, 2006 – Prosensa and Carolinas Medical Center announce their collaboration in the field of Duchenne Muscular Dystrophy. The collaboration will aim to develop an efficient systemic approach for Prosensa’s ‘smart therapy’. This ‘smart therapy’, acts by removing an unwanted segment of the faulty DMD gene product, thereby correcting the expression of the Dystrophin gene.
"We are extremely glad to initiate this collaboration. Dr. Lu has shown to be an expert in the field. We expect that, in addition to our collaboration with the LUMC, this will further enhance the potential of our technology, and thus form the basis for a viable cure for this terrible disease", says Gerard Platenburg, Prosensa's CEO.
“We have gained much experience in systemic delivery of RNA-based molecules in vivo. Now the moment has come to see whether our vision in combination with Prosensa’s expertise will lead to an improved approach to develop an efficient therapy” says Dr Qi Lu, Director of the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Carolinas Medical Center.
About DMD
DMD is a genetic lethal childhood’s disease with an incidence of approximately 1 in 3,500 newborn boys. Clinical signs of muscle weakness start as early as 2 years of age affecting all muscles. Treatment of patients with DMD to date primarily involves supportive treatments. No curative therapy, re-establishing the function of dystrophin, the protein that is lacking in DMD patients, is yet available.
About Carolinas Medical Center
Carolinas Medical Center (CMC) is the flagship facility of Carolinas HealthCare System which is the largest healthcare system in the Carolinas, and one of the largest public systems in the USA. As one of North Carolina's largest hospitals, it serves as the regional referral center for western North Carolina, South Carolina and all over the Southeast. The James G. Cannon Research Center (CRC), part of Carolinas HealthCare System located on the campus of Carolinas Medical Center. CRC focuses on translational and clinical research and development. In addition to the strong commitment of Carolinas HealthCare, a growing array of external grants and contracts, as well as endowments from charitable gifts and bequests, provide the financial base for an annual research budget of $19 million. Almost all of the efforts within Cannon Research Center are directed toward clinical applications. Research for experimental therapy to muscular dystrophy is one of the expanding areas in the CMC.
Press contacts for this press release
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Luc Dochez - Chief Business Officer - Prosensa
languages: English, Dutch, French
Timezone: Europe/Amsterdam
Phonetel phone_1: +31 (0) 71 33 22 085
Phonetel phone_2: +31 (0) 71 33 22 100
E-mail: l.dochez@prosensa.nl
