Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study-Reference-Cited by-同舟云学术

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Published:2014-10 Issue:10 Volume:13 Page:987-996
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

Voit Thomas,Topaloglu Haluk,Straub Volker,Muntoni Francesco,Deconinck Nicolas,Campion Giles,De Kimpe Sjef J,Eagle Michelle,Guglieri Michela,Hood Steve,Liefaard Lia,Lourbakos Afrodite,Morgan Allison,Nakielny Joanna,Quarcoo Naashika,Ricotti Valeria,Rolfe Katie,Servais Laurent,Wardell Claire,Wilson Rosamund,Wright Padraig,Kraus John E

Publisher

Elsevier BV

Subject

Clinical Neurology

Reference38 articles.

1. 195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14–16th December, 2012, Naarden, The Netherlands;Ellis;Neuromuscul Disord,2013

2. Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy;Mendell;Muscle Nerve,2013

3. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus;Monaco;Genomics,1988

4. Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy;Hoffman;N Engl J Med,1988

5. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

Cited by 262 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Therapeutic suppression ofTubb4arescues H-ABC leukodystrophy;2024-08-27

2. Splice-switching antisense oligonucleotides for pediatric neurological disorders;Frontiers in Molecular Neuroscience;2024-07-25

3. Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study;PLOS ONE;2024-07-10

4. RNA structure in alternative splicing regulation: from mechanism to therapy;Acta Biochimica et Biophysica Sinica;2024-07-01

5. Pharmacological Treatments and Therapeutic Targets in Muscle Dystrophies Generated by Alterations in Dystrophin-Associated Proteins;Medicina;2024-06-27