Literature DB >> 30033203

Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30-31 January 2017.

Volker Straub1, Eugenio Mercuri2.   

Abstract

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Year:  2018        PMID： 30033203     DOI： 10.1016/j.nmd.2018.05.013

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  8 in total

Review 1.  Advancements in magnetic resonance imaging-based biomarkers for muscular dystrophy.

Authors:  Doris G Leung
Journal:  Muscle Nerve       Date:  2019-05-14       Impact factor: 3.217

2.  Step Activity Monitoring in Boys with Duchenne Muscular Dystrophy and its Correlation with Magnetic Resonance Measures and Functional Performance.

Authors:  Kavya S Nair; Donovan J Lott; Sean C Forbes; Alison M Barnard; Rebecca J Willcocks; Claudia R Senesac; Michael J Daniels; Ann T Harrington; Gihan I Tennekoon; Kirsten Zilke; Erika L Finanger; Richard S Finkel; William D Rooney; Glenn A Walter; Krista Vandenborne
Journal:  J Neuromuscul Dis       Date:  2022

3.  Modeling disease trajectory in Duchenne muscular dystrophy.

Authors:  William D Rooney; Yosef A Berlow; William T Triplett; Sean C Forbes; Rebecca J Willcocks; Dah-Jyuu Wang; Ishu Arpan; Harneet Arora; Claudia Senesac; Donovan J Lott; Gihan Tennekoon; Richard Finkel; Barry S Russman; Erika L Finanger; Saptarshi Chakraborty; Elliott O'Brien; Brendan Moloney; Alison Barnard; H Lee Sweeney; Michael J Daniels; Glenn A Walter; Krista Vandenborne
Journal:  Neurology       Date:  2020-03-17       Impact factor: 9.910

4.  Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL.

Authors:  Philip A Powell; Jill Carlton; Donna Rowen; Fleur Chandler; Michela Guglieri; John E Brazier
Journal:  Neurology       Date:  2021-03-30       Impact factor: 9.910

Review 5.  First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases.

Authors:  Laurent Servais; Eric Camino; Aude Clement; Craig M McDonald; Jacek Lukawy; Linda P Lowes; Damien Eggenspieler; Francesca Cerreta; Paul Strijbos
Journal:  Digit Biomark       Date:  2021-08-05

Review 6.  The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.

Authors:  Shelagh M Szabo; Renna M Salhany; Alison Deighton; Meagan Harwood; Jean Mah; Katherine L Gooch
Journal:  Orphanet J Rare Dis       Date:  2021-05-22       Impact factor: 4.123

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.

Authors:  Laurent Servais; Eugenio Mercuri; Volker Straub; Michela Guglieri; Andreea M Seferian; Mariacristina Scoto; Daniela Leone; Erica Koenig; Navid Khan; Ashish Dugar; Xiaodong Wang; Baoguang Han; Dan Wang; Francesco Muntoni
Journal:  Nucleic Acid Ther       Date:  2021-11-17       Impact factor: 5.486

Review 8.  Mouse models for muscular dystrophies: an overview.

Authors:  Maaike van Putten; Erin M Lloyd; Jessica C de Greef; Vered Raz; Raffaella Willmann; Miranda D Grounds
Journal:  Dis Model Mech       Date:  2020-02-21       Impact factor: 5.758
  8 in total

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