Suchergebnisse - "Clemens, Paula R"

PubPharm (159)

1

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease : an open-label phase I/II study (ATB200-02)

enthalten in: Journal of neurology | 2024

von Byrne, B. | Schoser, B. | Kishnani, P. | +17

2

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease : a phase III open-label extension study (ATB200-07)

enthalten in: Journal of neurology | 2024

von Schoser, B. | Kishnani, P. | Bratkovic, D. | +81

3

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial

enthalten in: Neurology | 2024

von Dang, U. | Damsker, J. | Guglieri, M. | +41

4

Findings from the Longitudinal CINRG Becker Natural History Study

enthalten in: Journal of neuromuscular diseases | 2024

von Clemens, P. | Gordish-Dressman, H. | Niizawa, G. | +17

5

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

enthalten in: Journal of neurology | 2023

von Byrne, B. | Schoser, B. | Kishnani, P. | +17

6

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

7

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

8

Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy

enthalten in: Journal of neuromuscular diseases | 2023

von Fang, Y. | McDonald, C. | Clemens, P. | +5

9

Reply to F. Muntoni et al. : "In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

enthalten in: Journal of neuromuscular diseases | 2023

von Clemens, P. | Hoffman, E.

10

Antigen-driven T cell-macrophage interactions mediate the interface between innate and adaptive immunity in histidyl-tRNA synthetase-induced myositis

enthalten in: Frontiers in immunology | 2023

von Reay, D. | Tabib, T. | Wang, Y. | +6

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