194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8-10 December 2012, Naarden, The Netherlands

Neuromuscular Disorders

Volumn 23, Issue 11, 2013, Pages 934-944

  Aartsma Rus, Annemieke ^a   Muntoni, Francesco ^b  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CORTICOSTEROID; CREATINE KINASE; DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN; GELATINASE B; MESSENGER RNA PRECURSOR; MIPOMERSEN; MORPHOLINO OLIGONUCLEOTIDE; NANOPARTICLE; OLIGONUCLEOTIDE PHOSPHOROTHIOATE; PLACEBO; PRO 044; UNCLASSIFIED DRUG;

ARTICLE; BECKER MUSCULAR DYSTROPHY; CREATINE KINASE BLOOD LEVEL; DMD GENE; DOSAGE SCHEDULE COMPARISON; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DELIVERY SYSTEM; DRUG DISTRIBUTION; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG EXCRETION; DRUG HALF LIFE; DRUG INTERMITTENT THERAPY; DRUG MEGADOSE; DRUG METABOLISM; DRUG SAFETY; DRUG TARGETING; DRUG TISSUE LEVEL; DRUG TOLERABILITY; DRUG WITHDRAWAL; DUCHENNE MUSCULAR DYSTROPHY; ENZYME BLOOD LEVEL; EXON SKIPPING; FAMILIAL HYPERCHOLESTEROLEMIA; FLU LIKE SYNDROME; GENE DELETION; GENE MUTATION; HEART DISEASE; HUMAN; INJECTION SITE REACTION; INTERNATIONAL COOPERATION; LOW DRUG DOSE; MALIGNANT NEOPLASTIC DISEASE; MEDICAL RESEARCH; NETHERLANDS; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OPEN READING FRAME; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROTEINURIA; RESPIRATORY TRACT DISEASE; RNA SPLICING; SINGLE DRUG DOSE; SYSTEMIC THERAPY; THROMBOCYTOPENIA; WALKING;

EXONS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; NETHERLANDS; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84884980372     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2013.06.369     Document Type: Article

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