E-LIB Suche - Ergebnisse für: Clemens, Paula R

1

Findings from the Longitudinal CINRG Becker Natural History..:

Clemens, Paula R. ; Gordish-Dressman, Heather ; Niizawa, Gabriela...
Journal of Neuromuscular Diseases. 11 (2024) 1 - p. 201-212 , 2024

Link: https://doi.org/10.3233/..

2

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys Wit..:

Dang, Utkarsh J. ; Damsker, Jesse M. ; Guglieri, Michela...
Neurology. 102 (2024) 5 - p. , 2024

Link: https://doi.org/10.1212/..

3

Reply to F. Muntoni et al.: "In response to P.R. Clemens et..:

Clemens, Paula R. ; Hoffman, Eric P.
Journal of Neuromuscular Diseases. 10 (2023) 6 - p. 1155-1157 , 2023

Link: https://doi.org/10.3233/..

4

Efficacy and safety of avalglucosidase alfa in participants..:

Kishnani, Priya S. ; Díaz-Manera, Jordi ; Kushlaf, Hani...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107185 , 2023

Link: https://doi.org/10.1016/..

5

Long-term safety and efficacy of cipaglucosidase alfa plus ..:

Byrne, Barry J. ; Schoser, Benedikt ; Kishnani, Priya S....
Journal of Neurology. 271 (2023) 4 - p. 1787-1801 , 2023

Link: https://doi.org/10.1007/..

6

Modeling Early Heterogeneous Rates of Progression in Boys w..:

Fang, Yuan ; McDonald, Craig M. ; Clemens, Paula R....
Journal of Neuromuscular Diseases. 10 (2023) 3 - p. 349-364 , 2023

Link: https://doi.org/10.3233/..

7

Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Am..:

Mozaffar, Tahseen ; Schoser, Benedikt ; Kishnani, Priya...
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

Link: https://doi.org/10.1212/..

8

Long-term follow-up of cipaglucosidase alfa/miglustat in am..:

Byrne, Barry J. ; Schoser, Benedikt ; Kishnani, Priya...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107042 , 2023

Link: https://doi.org/10.1016/..

9

Efficacy and Safety of Viltolarsen in Boys With Duchenne Mu..:

Clemens, Paula R. ; Rao, Vamshi K. ; Connolly, Anne M....
Journal of Neuromuscular Diseases. 10 (2023) 3 - p. 439-447 , 2023

Link: https://doi.org/10.3233/..

10

Antigen-driven T cell-macrophage interactions mediate the i..:

Reay, Daniel P. ; Tabib, Tracy ; Wang, Ying...
Frontiers in Immunology. 14 (2023) - p. , 2023

Link: https://doi.org/10.3389/..

11

The avalglucosidase alfa phase 3 COMET trial in late-onset ..:

Kishnani, Priya ; Díaz-Manera, Jordi ; Kushlaf, Hani...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S66-S67 , 2022

Link: https://doi.org/10.1016/..

12

Long-Term Functional Efficacy and Safety of Viltolarsen in ..:

Clemens, Paula R. ; Rao, Vamshi K. ; Connolly, Anne M....
Journal of Neuromuscular Diseases. 9 (2022) 4 - p. 493-501 , 2022

Link: https://doi.org/10.3233/..

13

Safety and efficacy of avalglucosidase alfa versus alglucos..:

Diaz-Manera, Jordi ; Kishnani, Priya S ; Kushlaf, Hani...
The Lancet Neurology. 20 (2021) 12 - p. 1012-1026 , 2021

Link: https://doi.org/10.1016/..

14

Rasch Analysis of the Pediatric Quality of Life Inventory 4..:

Landfeldt, Erik ; Iff, Joel ; Henricson, Erik...
Value in Health. 24 (2021) 10 - p. 1490-1498 , 2021

Link: https://doi.org/10.1016/..

15

Concerns Regarding Therapeutic Implications of Very Low‐Lev..:

Hoffman, Eric P. ; Clemens, Paula R.
Annals of Neurology. 90 (2021) 1 - p. 176-176 , 2021

Link: https://doi.org/10.1002/..