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

1

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

Clemens, Paula R ; Rao, Vamshi K ; Connolly, Anne M...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10200237/. , 2023

Link: http://www.ncbi.nlm.nih...

2

Efficacy and Safety of Avalglucosidase Alfa in Patients wit..:

Straub, Volker ; Kostera-Pruszczyk, Anna ; Tammireddy, Swathi...
f6a1c166-a114-4db6-8661-5094b2fca2d6. , 2023

Link: https://doi.org/10.1001/..

3

Efficacy and Safety of Avalglucosidase Alfa in Patients Wit..:

Kishnani, Priya S ; Diaz-Manera, Jordi ; Toscano, Antonio...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10087094/. , 2023

Link: http://www.ncbi.nlm.nih...

4

Efficacy and Safety of Vamorolone in Duchenne Muscular Dyst..:

Mah, Jean K ; Clemens, Paula R ; Guglieri, Michela...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8790668/. , 2022

Link: http://www.ncbi.nlm.nih...

5

Efficacy and Safety of Vamorolone in Duchenne Muscular Dyst..:

Mah, Jean K ; Clemens, Paula R ; Guglieri, Michela...
https://discovery.ucl.ac.uk/id/eprint/10142870/1/Ridout_mah_2022_oi_211221_1642537152.18735.pdf. , 2022

Link: https://discovery.ucl.ac..

6

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

Hoffman, Eric P ; Clemens, Paula R
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8251539/. , 2021

Link: http://www.ncbi.nlm.nih...

7

Multi-Omics Identifies Circulating miRNA and Protein Biomar..:

Heier, Christopher R ; Zhang, Aiping ; Nguyen, Nhu Y...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7711540/. , 2020

Link: http://www.ncbi.nlm.nih...

8

The CINRG Becker Natural History Study: Baseline Characteri..:

Clemens, Paula R ; Niizawa, Gabriela ; Feng, Jia...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7520226/. , 2020

Link: http://www.ncbi.nlm.nih...

9

Exposure-Response Analysis of Vamorolone (VBP15) in Boys wi..:

Li, Xiaonan ; Conklin, Laurie S ; van den Anker, John...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7494537/. , 2020

Link: http://www.ncbi.nlm.nih...

10

Population Pharmacokinetics of Vamorolone (VBP15) in Health..:

Mavroudis, Panteleimon D ; van den Anker, John ; Conklin, Laurie S...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6548694/. , 2019

Link: http://www.ncbi.nlm.nih...

11

Vamorolone trial in Duchenne muscular dystrophy shows dose-..:

Hoffman, Eric P ; Schwartz, Benjamin D ; Mengle-Gaw, Laurel J...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7011869/. , 2019

Link: http://www.ncbi.nlm.nih...

12

GAA variants and phenotypes among 1,079 patients with Pompe..:

Reuser, Arnold J. J ; van der Ploeg, Ans T ; Chien, Yin‐Hsiu...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6852536/. , 2019

Link: http://www.ncbi.nlm.nih...

13

Diagnosis and management of Duchenne muscular dystrophy, pa..:

Birnkrant, David J ; Bushby, Katharine ; Bann, Carla M...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5869704/. , 2018

Link: http://www.ncbi.nlm.nih...

14

Phase IIa trial in Duchenne muscular dystrophy shows vamoro..:

Conklin, Laurie S ; Damsker, Jesse M ; Hoffman, Eric P...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6218284/. , 2018

Link: http://www.ncbi.nlm.nih...

15

A multinational study on motor function in early-onset FSHD:

Mah, Jean K ; Feng, Jia ; Jacobs, Marni B...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5894929/. , 2018

Link: http://www.ncbi.nlm.nih...