Parent Project Muscular Dystrophy is pleased to share this press release with all of our families.
PRESS RELEASE, May 10 2006
Prosensa and LUMC get approval for in-human trial for ‘smart therapy’ to cure Duchenne Muscular Dystrophy
On May 8, 2006 the Netherlands Central Committee on Research involving Human Subjects (CCMO) and the Medical Ethics Committee (IRB) of LUMC have granted Prosensa and LUMC permission to perform an exploratory study on the efficacy, safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide to restore production of the dystrophin, the protein defective in Duchenne Muscular Dystrophy (DMD) patients.
This very first in-human trial using antisense oligonucleotide, a ‘smart reagent’ removing an unwanted segment of the faulty DMD gene product, represents a major step toward the development of an effective therapy for DMD. It will provide an important proof-of-principle for future studies based on systemic, full-body, delivery.
"We are extremely glad to have permission for this study. We hope to start next month and expect to provide further information on the development in due time. We have all reasons to expect that this trial will prove the therapeutic 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 spent 5 years of laboratory and animal research and have successfully completed all the preparatory steps. Now the moment has come to see whether our vision to restore the defective protein has a chance of working in patients” say dr Judith van Deutekom, dr Jan Verschuuren and prof. dr Gertjan van Ommen, respectively lead investigators and Department head at the LUMC.
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 LUMC
Leiden University Medical Centre (LUMC) aims to play a leading role nationally and internationally, in the continuous improvement of health care quality. LUMC’s key tasks are research, patient care, and academic and post-academic medical education. It performs 11,500 daytime treatments and 19,000 hospital admissions yearly. It has 900 beds and employs 8700 people.
Parent Project Muscular Dystrophy (PPMD) is a not-for-profit organization founded in 1994 by parents and grandparents of children diagnosed with Duchenne and Becker Muscular Dystrophy. PPMD’s mission is to improve the treatment, quality of life and long term outlook for all individuals affected by Duchenne muscular dystrophy through research, education, advocacy and compassion.
PPMD has
made great strides in the DMD community by funding high impact research,
fighting to create standards of care, and providing up to date information to
families regarding DMD treatment and care.
It is the largest grassroots organization in the
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