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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Magazine article
Authors Nathalie M Goemans
Mar Tulinius
Johanna T van den Akker
Brigitte E Burm
Peter F Ekhart
Niki Heuvelmans
Tjadine Holling
Anneke A Janson
Gerard J Platenburg
Jessica A Sipkens
J M Ad Sitsen
Annemieke Aartsma-Rus
Gert-Jan B van Ommen
Gunnar Buyse
Niklas Darin
Jan J Verschuuren
Giles V Campion
Sjef J de Kimpe
Judith C van Deutekom
Published in The New England journal of medicine
Volume 364
Issue 16
Pages 1513-22
ISSN 1533-4406
Publication year 2011
Published at Institute of Clinical Sciences, Department of Pediatrics
Pages 1513-22
Language en
Links dx.doi.org/10.1056/NEJMoa1011367
Keywords Adolescent, Alternative Splicing, Child, Child, Preschool, Creatine Kinase, urine, Dose-Response Relationship, Drug, Dystrophin, genetics, metabolism, Exercise Test, Exons, Humans, Injections, Subcutaneous, Male, Muscle Strength, drug effects, Muscle, Skeletal, chemistry, metabolism, Muscular Dystrophy, Duchenne, drug therapy, genetics, Mutation, Oligonucleotides, administration & dosage, adverse effects, blood, therapeutic use, RNA, analysis
Subject categories Medical and Health Sciences

Abstract

Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne's muscular dystrophy with relevant mutations was previously reported to induce the skipping of exon 51 during pre-messenger RNA splicing of the dystrophin gene and to facilitate new dystrophin expression in muscle-fiber membranes. The present phase 1-2a study aimed to assess the safety, pharmacokinetics, and molecular and clinical effects of systemically administered PRO051.

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