Suchergebnisse - "McDonald, Craig M"

PubPharm (280)

51

Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2021

von Campbell, C. | McColl, E. | McDermott, M. | +45

52

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment

enthalten in: Muscle & nerve | 2021

von McDonald, C. | Wei, L. | Flanigan, K. | +5

53

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

enthalten in: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research | 2021

von Landfeldt, E. | Iff, J. | Henricson, E. | +37

54

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

enthalten in: Digital biomarkers | 2021

von Servais, L. | Camino, E. | Clement, A. | +6

55

Corrigendum to "Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy" [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502

enthalten in: Neuromuscular disorders : NMD | 2021

von Wagner, K. | Abdel-Hamid, H. | Mah, J. | +21

ErratumFor: Neuromuscul Disord. 2020 Jun;30(6):492-502. - PMID 32522498

56

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping : PROMOVI Trial

enthalten in: Journal of neuromuscular diseases | 2021

von McDonald, C. | Shieh, P. | Abdel-Hamid, H. | +12

57

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy : Results of the PolarisDMD Trial

enthalten in: Journal of neuromuscular diseases | 2021

von Finkel, R. | McDonald, C. | Lee Sweeney, H. | +24

58

Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

enthalten in: Journal of neuromuscular diseases | 2021

von Mendell, J. | Khan, N. | Sha, N. | +7

59

The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD

enthalten in: Journal of neuromuscular diseases | 2021

von Duong, T. | Canbek, J. | Birkmeier, M. | +7

60

(−)‐Epicatechin induces mitochondrial biogenesis and markers of muscle regeneration in adults with Becker muscular dystrophy

enthalten in: Muscle & Nerve | 2021

von McDonald, C. | Ramirez‐Sanchez, I. | Oskarsson, B. | +12