Literature DB >> 28636407

Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary.

Jonathan Finder1, Oscar Henry Mayer2, Daniel Sheehan3, Hemant Sawnani4, R Ted Abresch5, Joshua Benditt6, David J Birnkrant7,8, Tina Duong9, Erik Henricson5, Kathi Kinnett10, Craig M McDonald5,11, Anne M Connolly12.   

Abstract

Development of novel therapeutics for treatment of Duchenne muscular dystrophy (DMD) has led to clinical trials that include pulmonary endpoints that allow assessment of respiratory muscle status, especially in nonambulatory subjects. Parent Project Muscular Dystrophy (PPMD) convened a workshop in Bethesda, Maryland, on April 14 and 15, 2016, to summarize published respiratory data in DMD and give guidance to clinical researchers assessing the effect of interventions on pulmonary outcomes in DMD.

Entities:  

Keywords:  Duchenne muscular dystrophy; outcome; respiratory failure

Mesh：

Year:  2017        PMID： 28636407     DOI： 10.1164/rccm.201703-0507WS

Source DB:  PubMed          Journal:  Am J Respir Crit Care Med        ISSN： 1073-449X            Impact factor:   21.405


  7 in total

1.  Sarcolipin overexpression impairs myogenic differentiation in Duchenne muscular dystrophy.

Authors:  Nandita Niranjan; Satvik Mareedu; Yimin Tian; Kasun Kodippili; Nadezhda Fefelova; Antanina Voit; Lai-Hua Xie; Dongsheng Duan; Gopal J Babu
Journal:  Am J Physiol Cell Physiol       Date:  2019-07-31       Impact factor: 4.249

2.  Imaging respiratory muscle quality and function in Duchenne muscular dystrophy.

Authors:  Alison M Barnard; Donovan J Lott; Abhinandan Batra; William T Triplett; Sean C Forbes; Samuel L Riehl; Rebecca J Willcocks; Barbara K Smith; Krista Vandenborne; Glenn A Walter
Journal:  J Neurol       Date:  2019-07-26       Impact factor: 4.849

3.  Leveraging cardiac magnetic resonance imaging to assess skeletal muscle progression in Duchenne muscular dystrophy.

Authors:  Jacob A Kaslow; Andrew G Sokolow; Thomas Donnelly; Maciej S Buchowski; Bruce M Damon; Larry W Markham; W Bryan Burnette; Jonathan H Soslow
Journal:  Neuromuscul Disord       Date:  2022-02-04       Impact factor: 3.538

4.  Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History.

Authors:  T Bernard Kinane; Oscar H Mayer; Petra W Duda; Linda P Lowes; Stephanie L Moody; Jerry R Mendell
Journal:  J Neuromuscul Dis       Date:  2018

5.  Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy.

Authors:  Lindsay N Alfano; Jay S Charleston; Anne M Connolly; Linda Cripe; Cas Donoghue; Robert Dracker; Johannes Dworzak; Helen Eliopoulos; Diane E Frank; Sarah Lewis; Karin Lucas; Jessie Lynch; A J Milici; Amy Flynt; Emily Naughton; Louise R Rodino-Klapac; Zarife Sahenk; Frederick J Schnell; G David Young; Jerry R Mendell; Linda P Lowes
Journal:  Medicine (Baltimore)       Date:  2019-06       Impact factor: 1.817

6.  Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.

Authors:  Nathalie Goemans; James Signorovitch; Gautam Sajeev; Zhiwen Yao; Heather Gordish-Dressman; Craig M McDonald; Krista Vandenborne; Debra Miller; Susan J Ward; Eugenio Mercuri
Journal:  Neurology       Date:  2020-07-01       Impact factor: 9.910

7.  Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystroph.

Authors:  Gunnar M Buyse; Christian Rummey; Thomas Meier; Mika Leinonen; Thomas Voit; Craig M McDonald; Oscar H Mayer
Journal:  J Neuromuscul Dis       Date:  2018
  7 in total

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