Suchergebnisse - "McDonald, Craig M"

PubPharm (13)

1

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS) : a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

enthalten in: The Lancet. Neurology | 2024

von Mercuri, E. | Vilchez, J. | Boespflug-Tanguy, O. | +25

ErratumIn: Lancet Neurol. 2024 Apr 18;:. - PMID 38643778

2

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

enthalten in: Journal of comparative effectiveness research | 2023

von McDonald, C. | Marden, J. | Shieh, P. | +8

3

Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

enthalten in: Journal of comparative effectiveness research | 2022

von McDonald, C. | Muntoni, F. | Penematsa, V. | +36

4

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

5

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

6

Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2020

von Servais, L. | Straathof, C. | Schara, U. | +12

7

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

enthalten in: Neuromuscular disorders : NMD | 2018

von Goemans, N. | Mercuri, E. | Belousova, E. | +53

8

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

enthalten in: Journal of neuromuscular diseases | 2018

von Buyse, G. | Rummey, C. | Meier, T. | +4

9

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

enthalten in: Neurology | 2017

von Victor, R. | Sweeney, H. | Finkel, R. | +77

10

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD) : a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

enthalten in: Lancet (London, England) | 2017

von McDonald, C. | Campbell, C. | Torricelli, R. | +72

CommentIn: Lancet. 2017 Sep 23;390(10101):1467-1468. - PMID 28728957