Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

Ryan S. Paquin; Ryan Fischer; Carol Mansfield; Brennan Mange; Katherine Beaverson; Annie Ganot; Amy Strong Martin; Carl Morris; Colin Rensch; Valeria Ricotti; Leo J. Russo; Alesia Sadosky; Edward C. Smith; Holly L. Peay

doi:10.1186/s13023-019-1069-6

Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

Orphanet Journal of Rare Diseases ◽

10.1186/s13023-019-1069-6 ◽

2019 ◽

Vol 14 (1) ◽

Cited By ~ 2

Author(s):

Ryan S. Paquin ◽

Ryan Fischer ◽

Carol Mansfield ◽

Brennan Mange ◽

Katherine Beaverson ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Early Phase ◽

Adult Patients ◽

Best Worst Scaling

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Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy

10.21236/ada613171 ◽

2014 ◽

Author(s):

Barry J. Byrne

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Respiratory Insufficiency

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Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Molecular Therapy — Methods & Clinical Development ◽

10.1016/j.omtm.2020.06.016 ◽

2020 ◽

Vol 18 ◽

pp. 415-421 ◽

Cited By ~ 3

Author(s):

Virginie Mariot ◽

Caroline Le Guiner ◽

Inès Barthélémy ◽

Marie Montus ◽

Stéphane Blot ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy

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92. Safety and Histological Signs of Efficacy Following Systemic AAV9-CMV-Microutrophin Gene Therapy in a Canine Model for Duchenne Muscular Dystrophy

Molecular Therapy ◽

10.1016/s1525-0016(16)34427-6 ◽

2013 ◽

Vol 21 ◽

pp. S38

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Canine Model

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Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling

Journal of Genetic Counseling ◽

10.1007/s10897-015-9872-2 ◽

2015 ◽

Vol 25 (2) ◽

pp. 305-313 ◽

Cited By ~ 16

Author(s):

Holly Landrum Peay ◽

I. L. Hollin ◽

J. F. P. Bridges

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Best Worst Scaling

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Long-term follow-up of dysphagia in adult patients with Duchenne muscular dystrophy

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2018.06.004 ◽

2018 ◽

Vol 22 (5) ◽

pp. 786-790 ◽

Cited By ~ 2

Author(s):

Yuka Yamada ◽

Michiyuki Kawakami ◽

Ayako Wada ◽

Shogo Fukui ◽

Koshiro Haruyama ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Adult Patients ◽

Long Term Follow Up

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Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

Methods in Molecular Biology - Muscle Gene Therapy ◽

10.1007/978-1-61737-982-6_2 ◽

2010 ◽

pp. 21-37 ◽

Cited By ~ 12

Author(s):

Takis Athanasopoulos ◽

Helen Foster ◽

Keith Foster ◽

George Dickson

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Codon Optimization ◽

Duchenne Muscular Dystrophy Gene

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Dystrophin mRNA Processing in the Canine Homologue of Duchenne Muscular Dystrophy: An Authentic Model for Gene Therapy

Research and Perspectives in Neurosciences - Gene Transfer and Therapy in the Nervous System ◽

10.1007/978-3-642-84842-1_12 ◽

1992 ◽

pp. 146-157

Author(s):

N. J. H. Sharp ◽

J. N. Kornegay ◽

S. D. van Camp ◽

M. H. Herbstreith ◽

S. L. Secore ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Mrna Processing

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790. Evaluation of Immune Responses to Dystrophin in Humanized mdx HLA-A*0201 Dystrophic Mice: Application in Duchenne Muscular Dystrophy Gene Therapy after U7 snRNA-Mediated Exon Skipping

Molecular Therapy ◽

10.1016/j.ymthe.2005.07.328 ◽

2005 ◽

Vol 11 ◽

pp. S307 ◽

Cited By ~ 1

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Immune Responses ◽

Exon Skipping ◽

Duchenne Muscular Dystrophy Gene ◽

Dystrophic Mice

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AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

Journal of Molecular and Cellular Cardiology ◽

10.1016/j.yjmcc.2012.05.002 ◽

2012 ◽

Vol 53 (2) ◽

pp. 217-222 ◽

Cited By ~ 44

Author(s):

Brian Bostick ◽

Jin-Hong Shin ◽

Yongping Yue ◽

Nalinda B. Wasala ◽

Yi Lai ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Myocardial Fibrosis ◽

Cardiac Death ◽

Dystrophin Gene ◽

Mdx Mice ◽

Stage Model ◽

End Stage ◽

Duchenne Muscular Dystrophy Cardiomyopathy

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1131. A Variant of Human Dihydrofolate Reductase Can Be Used To Select Myoblasts for Ex Vivo Gene Therapy of Duchenne Muscular Dystrophy

Molecular Therapy ◽

10.1016/s1525-0016(16)43961-4 ◽

2002 ◽

Vol 5 (5) ◽

pp. S367-S368

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Dihydrofolate Reductase ◽

Ex Vivo ◽

Ex Vivo Gene Therapy ◽

Human Dihydrofolate Reductase

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