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Systemic administra...
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
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Goemans, Nathalie M (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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van den Akker, Johanna T (author)
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Burm, Brigitte E (author)
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Ekhart, Peter F (author)
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Heuvelmans, Niki (author)
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Holling, Tjadine (author)
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Janson, Anneke A (author)
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Platenburg, Gerard J (author)
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Sipkens, Jessica A (author)
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Sitsen, J M Ad (author)
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Aartsma-Rus, Annemieke (author)
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van Ommen, Gert-Jan B (author)
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Buyse, Gunnar (author)
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- Darin, Niklas, 1964 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Verschuuren, Jan J (author)
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Campion, Giles V (author)
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de Kimpe, Sjef J (author)
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van Deutekom, Judith C (author)
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(creator_code:org_t)
- 2011
- 2011
- English.
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In: The New England journal of medicine. - 1533-4406. ; 364:16, s. 1513-22
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https://doi.org/10.1...
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Abstract
Subject headings
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- 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.
Keyword
- 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
Publication and Content Type
- vet (subject category)
- art (subject category)
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- By the author/editor
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Goemans, Nathali ...
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Tulinius, Mar, 1 ...
-
van den Akker, J ...
-
Burm, Brigitte E
-
Ekhart, Peter F
-
Heuvelmans, Niki
-
show more...
-
Holling, Tjadine
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Janson, Anneke A
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Platenburg, Gera ...
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Sipkens, Jessica ...
-
Sitsen, J M Ad
-
Aartsma-Rus, Ann ...
-
van Ommen, Gert- ...
-
Buyse, Gunnar
-
Darin, Niklas, 1 ...
-
Verschuuren, Jan ...
-
Campion, Giles V
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de Kimpe, Sjef J
-
van Deutekom, Ju ...
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show less...
- Articles in the publication
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The New England ...
- By the university
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University of Gothenburg