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Research article

First published 2017

Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach

Nicolas Wein, Adeline Vulin, Andrew R. Findlay [...]

Journal of Neuromuscular Diseases

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Research article

First published 1998-8

A Theoretical and Experimental Study of the Fragment Ion of m/z 97 Obtained from the Dimethyl Disulfide Derivative of Unsaturated Ethyl Esters

C. Pepe, H. Sayer, J. Dagaut

European Journal of Mass Spectrometry

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Research article

First published 2003-11

Dystrophin Delivery in Dystrophin-Deficient DMD mdx Skeletal Muscle by Isogenic Muscle-Derived Stem Cell Transplantation

Makoto Ikezawa, Baohong Cao, Zhuqing Qu [...]

Human Gene Therapy

Retricted access

Research article

First published 1999-8

Letter: Determination of Positions of Double Bonds in Conjugated Alkadienes by Means of Gas Chromatography/Mass Spectrometry of Dimethyl Disulfide Derivatives

C. Pepe, H. Sayer, P. Bacelon

European Journal of Mass Spectrometry

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Research article

First published 2020-02

Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts

Jinhong Meng, Nathan Paul Sweeney, Bruno Doreste [...]

Human Gene Therapy

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Research article

First published 1984-2

The Dirichlet Multinomial Distribution as a Magazine Exposure Model

John D. Leckenby, Shizue Kishi

Journal of Marketing Research

Open access

Research article

First published 2020-7

Mobile phone-based e-diary for assessment and enhancement of medications adherence among patients with multiple sclerosis

Daniel Golan, Smadar Sagiv, Lea Glass-Marmor [...]

Multiple Sclerosis Journal – Experimental,Translational and Clinical

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Review article

First published 2016-10

Duchenne Muscular Dystrophy Myogenic Cells from Urine-Derived Stem Cells Recapitulate the Dystrophin Genotype and Phenotype

Maria Sofia Falzarano, Domenico D'Amario, Andrea Siracusano [...]

Human Gene Therapy

Open access

Review article

First published 2023

Cardiac therapies for Duchenne muscular dystrophy

Md Nur Ahad Shah, Toshifumi Yokota

Therapeutic Advances in Neurological Disorders

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Research article

First published 2022-01-04

Differentiation of Pediatric-Onset Duchenne and Becker Muscular Dystrophy Subphenotypes Using Data from the Muscular Dystrophy Surveillance Tracking and Research Network (MD STAR net )

Jennifer G. Andrews, Molly M. Lamb, Kristin M. Conway [...]

Journal of Neuromuscular Diseases

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