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

1

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/..

2

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/..

3

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/..

4

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/..

5

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/..

6

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/..

7

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/..

8

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/..

9

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/..

10

Exposure‐Response Analysis of Vamorolone (VBP15) in Boys Wi..:

Li, Xiaonan ; Conklin, Laurie S. ; den Anker, John...
The Journal of Clinical Pharmacology. 60 (2020) 10 - p. 1385-1396 , 2020

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

11

Efficacy and safety of vamorolone in Duchenne muscular dyst..:

Smith, Edward C. ; Conklin, Laurie S. ; Hoffman, Eric P....
PLOS Medicine. 17 (2020) 9 - p. e1003222 , 2020

Link: https://doi.org/10.1371/..

12

TheCINRGBecker Natural History Study: Baseline characterist..:

Clemens, Paula R. ; Niizawa, Gabriela ; Feng, Jia...
Muscle & Nerve. 62 (2020) 3 - p. 369-376 , 2020

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

13

Serum biomarkers associated with baseline clinical severity..:

Dang, Utkarsh J ; Ziemba, Michael ; Clemens, Paula R...
Human Molecular Genetics. 29 (2020) 15 - p. 2481-2495 , 2020

Link: https://doi.org/10.1093/..

14

Disease-specific and glucocorticoid-responsive serum biomar..:

Hathout, Yetrib ; Liang, Chen ; Ogundele, Michael...
Scientific Reports. 9 (2019) 1 - p. , 2019

Link: https://doi.org/10.1038/..

15

Preliminary patient-reported outcomes and safety of advance..:

Schoser, Benedikt ; Bratkovic, Drago ; Byrne, Barry J....
Molecular Genetics and Metabolism. 126 (2019) 2 - p. S132-S133 , 2019

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