Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial-Reference-Cited by-同舟云学术

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Published:2022-02-01 Issue:1 Volume:32 Page:29-39
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Servais Laurent¹²³,Mercuri Eugenio⁴⁵,Straub Volker⁶,Guglieri Michela⁶,Seferian Andreea M.¹,Scoto Mariacristina⁷⁸,Leone Daniela⁵,Koenig Erica⁹,Khan Navid⁹,Dugar Ashish⁹,Wang Xiaodong⁹,Han Baoguang⁹,Wang Dan⁹,Muntoni Francesco⁷⁸^ORCID,Brusa Chiara,Antonaci Laura,Brogna Claudia,Merli Laura,Monforte Mauro,Norcia Giulia,Pane Marika,Ferrantini Gloria,Dickson George,Morgan Jennifer,Sardone Valentina,Akana Cody,Charleston Jay S.,Desjardins Cody A.,El-Husayni Saleh H.,Frank Diane E.,Schnell Frederick J.,

Affiliation:

1. I-Motion Institute, Hôpital Armand Trousseau, Paris, France.

2. Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium.

3. MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom.

4. Pediatric Neurology Unit, Università Cattolica del Sacro Cuore Roma, Rome, Italy.

5. Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy.

6. John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

7. Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, United Kingdom.

8. National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom.

9. Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2021.0043

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1. DMDgenotypes and loss of ambulation in the CINRG Duchenne Natural History Study

2. Current and emerging treatment strategies for Duchenne muscular dystrophy