Suchergebnisse - "McDonald, Craig M"

PubPharm (26)

1

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

2

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy : A 30-Month Nonrandomized Controlled Open-Label Extension Trial

enthalten in: JAMA network open | 2022

von Mah, J. | Clemens, P. | Guglieri, M. | +125

3

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

4

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

enthalten in: Neurology | 2020

von Goemans, N. | Signorovitch, J. | Sajeev, G. | +92

5

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping : A Phase 2 Randomized Clinical Trial

enthalten in: JAMA neurology | 2020

von Clemens, P. | Rao, V. | Connolly, A. | +11

ErratumIn: JAMA Neurol. 2020 Aug 1;77(8):1040. - PMID 32539096

6

TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy

enthalten in: European journal of human genetics : EJHG | 2020

von Spitali, P. | Zaharieva, I. | Bohringer, S. | +59

7

Genetic modifiers of respiratory function in Duchenne muscular dystrophy

enthalten in: Annals of clinical and translational neurology | 2020

von Bello, L. | D'Angelo, G. | Villa, M. | +38

8

Safety and effectiveness of ataluren : comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

enthalten in: Journal of comparative effectiveness research | 2020

von Mercuri, E. | Muntoni, F. | Osorio, A. | +14

CommentIn: Pediatr Neurol Briefs. 2020 Dec 04;34:12. - PMID 33304086

9

Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy : Long-term natural history with and without glucocorticoids

enthalten in: Neuromuscular disorders : NMD | 2018

von McDonald, C. | Gordish-Dressman, H. | Henricson, E. | +45

10

Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy

enthalten in: Muscle & nerve | 2018

von McDonald, C.

CommentOn: Muscle Nerve. 2018 Nov;58(5):631-638. - PMID 29742798