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1 . Academic Journal
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
저자
by
Dominic Scaglioni
;
Francesco Catapano
;
Matthew Ellis
;
Silvia Torelli
;
Darren Chambers
, et al.
소스
Acta Neuropathologica Communications, Vol 9, Iss 1, Pp 1-17 (2021)
Open Access (BioMed Central)
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7 .
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
저자
by
Ashish Dugar
;
Eugenio Mercuri
;
Xiaodong Wang
;
Volker Straub
;
Baoguang Han
, et al.
소스
Nucleic Acid Therapeutics
Nucleic Acid Therapeutics, In press, ⟨10.1089/nat.2021.0043⟩
Nucleic Acid Therapeutics, Mary Ann Liebert, Inc. publishers, In press, ⟨10.1089/nat.2021.0043⟩
Open Access (OpenAIRE)
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7 .
Safety, tolerability, and pharmacokinetics of casimersen in patients with
D
uchenne muscular dystrophy amenable to exon
45
skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial
저자
by
Erica Koenig
;
Nancy L. Kuntz
;
Lilly East
;
Kathryn R. Wagner
;
Perry B. Shieh
, et al.
소스
Muscle & Nerve
. 64:285-292
Full Text (Wiley)
Open Access (OpenAIRE)
Web of Science
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7 .
eP212: Safety, β-Sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4
저자
by
Erin O’Rourke
;
Louise Rodino-Klapac
;
Eric Pozsgai
;
Sarah Lewis
;
Danielle Griffin
, et al.
소스
Genetics in Medicine
. 24:S132-S133
Full Text (ScienceDirect O/A)
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7 .
Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054
저자
by
Jason Aldred
;
Natalia Penner
;
Pepinsky R Blake
;
Ira J. Goodman
;
Erica Koenig
, et al.
소스
Movement Disorders
Full Text (Wiley)
Open Access (OpenAIRE)
Web of Science
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7 .
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial
저자
by
Craig M. McDonald
;
Kathryn R. Wagner
;
Erica Koenig
;
Jyoti Malhotra
;
Navid Khan
, et al.
소스
Journal of Neuromuscular Diseases
Open Access (OpenAIRE)
요약보기
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7 .
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
저자
by
F. Catapano
;
Jennifer E. Morgan
;
Caroline Sewry
;
Matthew Ellis
;
D. Scaglioni
, et al.
소스
Acta Neuropathologica Communications
Acta Neuropathologica Communications, Vol 9, Iss 1, Pp 1-17 (2021)
Open Access (BioMed Central)
Open Access (OpenAIRE)
Scopus
JCR 저널정보
요약보기
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E-Mail
EndNote
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7 .
Casimersen treatment in eligible patients with Duchenne muscular dystrophy: safety, tolerability, and pharmacokinetics over 144 weeks of treatment
저자
by
Kathryn R. Wagner
;
Nancy L. Kuntz
;
Perry B. Shieh
;
Erin O’Rourke
;
Sameer Upadhyay
, et al.
소스
Molecular Genetics and Metabolism
. 132:S101-S102
Web of Science
Scopus
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