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On May 8, 2006 the Netherlands Central Committee on Research involving Human Subjects (CCMO) and the Medical Ethics Committee (IRB) of LUMC gave their permission to be performed 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.