Literature DB >> 27997035

Regarding "Eteplirsen for the treatment of Duchenne muscular dystrophy".

Ellis F Unger1, Robert M Califf1.   

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Year:  2017        PMID： 27997035     DOI： 10.1002/ana.24842

Source DB:  PubMed          Journal:  Ann Neurol        ISSN： 0364-5134            Impact factor:   10.422


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  12 in total

Review 1.  Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.

Authors:  Luca Bello; Elena Pegoraro
Journal:  Acta Myol       Date:  2016-12

2.  Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy.

Authors:  Ilene L Hollin; Holly Peay; Ryan Fischer; Ellen M Janssen; John F P Bridges
Journal:  Qual Life Res       Date:  2018-05-26       Impact factor: 4.147

3.  In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

Authors:  Mona El Refaey; Li Xu; Yandi Gao; Benjamin D Canan; T M Ayodele Adesanya; Sarah C Warner; Keiko Akagi; David E Symer; Peter J Mohler; Jianjie Ma; Paul M L Janssen; Renzhi Han
Journal:  Circ Res       Date:  2017-08-08       Impact factor: 17.367

Review 4.  Delivery of Oligonucleotide Therapeutics: Chemical Modifications, Lipid Nanoparticles, and Extracellular Vesicles.

Authors:  Jeremy P Bost; Hanna Barriga; Margaret N Holme; Audrey Gallud; Marco Maugeri; Dhanu Gupta; Taavi Lehto; Hadi Valadi; Elin K Esbjörner; Molly M Stevens; Samir El-Andaloussi
Journal:  ACS Nano       Date:  2021-09-10       Impact factor: 15.881

5.  Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost.

Authors:  Marinos C Dalakas
Journal:  Ther Adv Neurol Disord       Date:  2017-06-28       Impact factor: 6.570

Review 6.  2'-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping.

Authors:  Tomoko Lee; Hiroyuki Awano; Mariko Yagi; Masaaki Matsumoto; Nobuaki Watanabe; Ryoya Goda; Makoto Koizumi; Yasuhiro Takeshima; Masafumi Matsuo
Journal:  Genes (Basel)       Date:  2017-02-10       Impact factor: 4.096

7.  Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse.

Authors:  Derek W Wang; Ekaterina I Mokhonova; Genevieve C Kendall; Diana Becerra; Yalda B Naeini; Rita M Cantor; Melissa J Spencer; Stanley F Nelson; M Carrie Miceli
Journal:  Mol Ther Nucleic Acids       Date:  2018-02-13       Impact factor: 8.886

8.  Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.

Authors:  Holly Landrum Peay; Ryan Fischer; Janice P Tzeng; Sharon E Hesterlee; Carl Morris; Amy Strong Martin; Colin Rensch; Edward Smith; Valeria Ricotti; Katherine Beaverson; Hannah Wand; Carol Mansfield
Journal:  PLoS One       Date:  2019-05-01       Impact factor: 3.240

9.  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.

Authors:  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
Journal:  Orphanet J Rare Dis       Date:  2019-05-09       Impact factor: 4.123

Review 10.  Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.

Authors:  Xiulong Shen; David R Corey
Journal:  Nucleic Acids Res       Date:  2018-02-28       Impact factor: 16.971
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