Suchergebnisse - "Ciafaloni, Emma"

PubPharm (27)

1

Safety and efficacy of arimoclomol for inclusion body myositis : a multicentre, randomised, double-blind, placebo-controlled trial

enthalten in: The Lancet. Neurology | 2023

von Machado, P. | McDermott, M. | Blaettler, T. | +91

CommentIn: Lancet Neurol. 2023 Oct;22(10):873-874. - PMID 37739563

2

Newborn screening for Duchenne muscular dystrophy : A two-year pilot study

enthalten in: Annals of clinical and translational neurology | 2023

von Tavakoli, N. | Gruber, D. | Armstrong, N. | +24

3

Time to diagnosis of Duchenne muscular dystrophy remains unchanged : Findings from the Muscular Dystrophy Surveillance, Tracking, and Research Network, 2000-2015

enthalten in: Muscle & nerve | 2022

von Thomas, S. | Conway, K. | Fapo, O. | +9

CommentIn: Muscle Nerve. 2022 Aug;66(2):116-117. - PMID 35560237

4

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

enthalten in: Journal of comparative effectiveness research | 2021

von Shieh, P. | Elfring, G. | Trifillis, P. | +111

5

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

6

Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis

enthalten in: Muscle & nerve | 2021

von Sansone, V. | Johnson, N. | Hanna, M. | +5

7

The Spinal Muscular Atrophy Health Index : A novel outcome for measuring how a patient feels and functions

enthalten in: Muscle & nerve | 2021

von Zizzi, C. | Luebbe, E. | Mongiovi, P. | +10

8

Spinal muscular atrophy and Farber disease due to ASAH1 variants : A case report

enthalten in: American journal of medical genetics. Part A | 2020

von Lee, B. | Mongiovi, P. | Levade, T. | +3

9

Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2020

von Kay, D. | Stevens, C. | Parker, A. | +15

CommentIn: Genet Med. 2020 Nov;22(11):1913-1914. - PMID 32601389

10

Minimal manifestation status and prednisone withdrawal in the MGTX trial

enthalten in: Neurology | 2020

von Lee, I. | Kuo, H. | Aban, I. | +144