Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study

The Lancet

Volumn 378, Issue 9791, 2011, Pages 595-605

  Cirak, Sebahattin ^a   Arechavala Gomeza, Virginia ^a   Guglieri, Michela ^b   Feng, Lucy ^a   Torelli, Silvia ^a   Anthony, Karen ^a   Abbs, Stephen ^c   Garralda, Maria Elena ^d   Bourke, John ^b   Wells, Dominic J ^e   Dickson, George ^f   Wood, Matthew Ja ^g   Wilton, Steve D ^h   Straub, Volker ^b   Kole, Ryszard ⁱ   Shrewsbury, Stephen B ⁱ   Sewry, Caroline ^a,j   Morgan, Jennifer E ^a   Bushby, Kate ^b   Muntoni, Francesco ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b NEWCASTLE UNIVERSITY   (United Kingdom)

^c GUY S HOSPITAL   (United Kingdom)

^d IMPERIAL COLLEGE LONDON   (United Kingdom)

^e ROYAL VETERINARY COLLEGE   (United Kingdom)

^f UNIVERSITY OF LONDON   (United Kingdom)

^g UNIVERSITY OF OXFORD   (United Kingdom)

^h UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

ⁱ AVI BioPharma   (United States)

^j ROBERT JONES AND AGNES HUNT ORTHOPAEDIC HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA SARCOGLYCAN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; BISOPROLOL; CALCIUM; CORTICOSTEROID; DEFLAZACORT; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; DYSTROPHIN; ETEPLIRSEN; LISINOPRIL; NEURONAL NITRIC OXIDE SYNTHASE; OMEPRAZOLE; PERINDOPRIL; PREDNISOLONE; RANITIDINE; RISEDRONIC ACID; VITAMIN D;

ADOLESCENT; ARTICLE; BONE MARROW; CARDIOMYOPATHY; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CORTICOSTEROID THERAPY; CYTOTOXIC T LYMPHOCYTE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FEMALE; FLUORESCENCE; FORCED VITAL CAPACITY; HUMAN; IMMUNOHISTOCHEMISTRY; INFLAMMATORY INFILTRATE; KIDNEY FUNCTION; LUNG FUNCTION; MALE; MUSCLE BIOPSY; OPEN STUDY; PHASE 2 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; SARCOLEMMA; SCHOOL CHILD; SIDE EFFECT; SINUS TACHYCARDIA; TREATMENT RESPONSE; UNSPECIFIED SIDE EFFECT; WESTERN BLOTTING;

EID: 80051690306     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(11)60756-3     Document Type: Article

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