Suchergebnisse - "Zaidman, Craig M"

PubPharm (102)

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

Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy

enthalten in: Journal of neuromuscular diseases | 2024

von Zaidman, C. | Goedeker, N. | Aqul, A. | +11

3

Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy

enthalten in: Pediatric neurology | 2024

von Mendell, J. | Proud, C. | Zaidman, C. | +8

4

Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy : 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)

enthalten in: Annals of neurology | 2023

von Zaidman, C. | Proud, C. | McDonald, C. | +15

5

Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular Dystrophy

enthalten in: Journal of neuromuscular diseases | 2023

von Connolly, A. | Zaidman, C. | Brandsema, J. | +6

6

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy : Results From the Phase 2, Open-Label, 4-Year Extension Study

enthalten in: Journal of neuromuscular diseases | 2023

von Clemens, P. | Rao, V. | Connolly, A. | +8

7

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

enthalten in: Therapeutic advances in neurological disorders | 2023

von Goedeker, N. | Dharia, S. | Griffin, D. | +7

8

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy : Phase I Ascending-Dose Study (STRONG)

enthalten in: Journal of neuromuscular diseases | 2023

von Finkel, R. | Darras, B. | Mendell, J. | +14

9

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

10

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

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