Suchergebnisse - "Shieh, Perry B"

PubPharm (9)

1

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks : A Phase 3 Randomized Clinical Trial

enthalten in: JAMA neurology | 2023

von Kishnani, P. | Diaz-Manera, J. | Toscano, A. | +125

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

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1 : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +17

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

4

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +18

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

5

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE) : an open-label, single-arm, multicentre, phase 3 trial

enthalten in: The Lancet. Neurology | 2021

von Day, J. | Finkel, R. | Chiriboga, C. | +18

CommentIn: Lancet Neurol. 2021 Apr;20(4):251-252. - PMID 33743228

6

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

7

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

enthalten in: The New England journal of medicine | 2018

von Mercuri, E. | Darras, B. | Chiriboga, C. | +121

CommentIn: Nat Rev Neurol. 2018 Apr;14(4):196. - PMID 29497154

8

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

9

Amifampridine phosphate (Firdapse(®)) is effective and safe in a phase 3 clinical trial in LEMS

enthalten in: Muscle & nerve | 2016

von Oh, S. | Shcherbakova, N. | Kostera-Pruszczyk, A. | +30

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