E-LIB Suche - Ergebnisse für: Cox, Gerald F

1

Clinical outcome assessments of disease burden and progress..:

Kissell, Julie ; Rochmann, Camille ; Minini, Pascal...
Molecular Genetics and Metabolism. 142 (2024) 3 - p. 108512 , 2024

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

2

Improved gene therapy for spinal muscular atrophy in mice u..:

Xie, Qing ; Chen, Xiupeng ; Ma, Hong...
EMBO Molecular Medicine. 16 (2024) 4 - p. 945-965 , 2024

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

3

Neuropsychological endpoints for clinical trials in methylm..:

Chapman, Kimberly A. ; MacEachern, Devon ; Cox, Gerald F....
Molecular Genetics and Metabolism Reports. 34 (2023) - p. 100953 , 2023

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

4

CAN008 prolongs overall survival in patients with newly dia..:

Chang, Ian Yi-Feng ; Tsai, Hong-Chieh ; Chen, Chia-Hua...
Biomedical Journal. , 2023

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

5

Plasma glucosylsphingosine correlations with baseline disea..:

Peterschmitt, M. Judith ; Foster, Meredith C. ; Ji, Allena J...
Molecular Genetics and Metabolism. 138 (2023) 3 - p. 107527 , 2023

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

6

Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo..:

Hastings, Caroline ; Liu, Benny ; Hurst, Bryan..
Molecular Genetics and Metabolism. 137 (2022) 4 - p. 309-319 , 2022

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

7

Effects of paroxetine, ketoconazole, and rifampin on the me..:

Vu, Lucie ; Cox, Gerald F. ; Ibrahim, Jennifer...
Molecular Genetics and Metabolism Reports. 22 (2020) - p. 100552 , 2020

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

8

How a concentration-effect analysis of data from the eliglu..:

Ruskin, Jeremy N. ; Ortemann-Renon, Catherine ; Msihid, Jerome...
Molecular Genetics and Metabolism. 131 (2020) 1-2 - p. 211-218 , 2020

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

9

Patient and caregiver perspectives on burden of disease man..:

Lyn, Nicole ; Pulikottil-Jacob, Ruth ; Rochmann, Camille...
Orphanet Journal of Rare Diseases. 15 (2020) 1 - p. , 2020

Link: https://doi.org/10.1186/..

10

Alternative genomic diagnoses for individuals with a clinic..:

Dyment, David A. ; O'Donnell‐Luria, Anne ; Agrawal, Pankaj B....
American Journal of Medical Genetics Part A. 185 (2020) 1 - p. 119-133 , 2020

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

11

Addendum to Letter to the Editor: Safety, efficacy, and aut..:

Mistry, Pramod K. ; Balwani, Manisha ; Baris, Hagit N....
Blood Cells, Molecules, and Diseases. 77 (2019) - p. 101-102 , 2019

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

12

Corrigendum to "Cause of death in patients with chronic vis..:

Cassiman, David ; Packman, Seymour ; Bembi, Bruno...
Molecular Genetics and Metabolism. 125 (2018) 4 - p. 360 , 2018

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

13

The art and science of choosing efficacy endpoints for rare..:

Cox, Gerald F.
American Journal of Medical Genetics Part A. 176 (2018) 4 - p. 759-772 , 2018

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

14

A pooled analysis of adverse events in 393 adults with Gauc..:

Peterschmitt, M. Judith ; Cox, Gerald F. ; Ibrahim, Jennifer...
Blood Cells, Molecules, and Diseases. 68 (2018) - p. 185-191 , 2018

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

15

Safety, efficacy, and authorization of eliglustat as a firs..:

Mistry, Pramod K. ; Balwani, Manisha ; Baris, Hagit N....
Blood Cells, Molecules, and Diseases. 71 (2018) - p. 71-74 , 2018

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