E-LIB Suche - Ergebnisse für: Dali, Christine í

1

Evaluation of the long term effect of arimoclomol in NPC:

Patterson, Marc ; Dali, Christine i ; Mengel, Eugen
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107260 , 2023

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

2

Association between NPC severity score domains and correspo..:

Dali, Christine i ; Symonds, Tara ; Doll, Helen...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107066 , 2023

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

3

Effects of sulfatide on peripheral nerves in metachromatic ..:

Farah, Mohamed H. ; Dali, Christine í ; Groeschel, Samuel...
Annals of Clinical and Translational Neurology. 11 (2023) 2 - p. 328-341 , 2023

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

4

Arimoclomol reduces levels of biomarkers of lipid burden in..:

Andersen, Lene ; Petersen, Nikolaj H. ; Ingemann, Linda...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S18 , 2022

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

5

Persistent effect of arimoclomol in patients with Niemann-P..:

Patterson, Marc ; Mengel, Eugen ; Da Rio, Rosalia M....
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S94 , 2022

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

6

Effects of sulfatide accumulation on peripheral nerves in m..:

Krarup, Christian ; Dali, Christine í ; Groeschel, Samuel...
Neurology. 98 (2022) 18_supplement - p. , 2022

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

7

Correction to: Clinical disease progression and biomarkers ..:

Mengel, Eugen ; Bembi, Bruno ; del Toro, Mireia...
Orphanet Journal of Rare Diseases. 16 (2021) 1 - p. , 2021

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

8

Impacts and burden of Niemann-Pick disease type C: A patien..:

Mengel, Eugen ; Patterson, Marc ; Chladek, Michael...
Molecular Genetics and Metabolism. 132 (2021) 2 - p. S71 , 2021

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

9

Rescue of NPC1 protein and effect on biomarkers by arimoclo..:

Ingemann, Linda ; Petersen, Nikolaj Havnsøe Torp ; Koustrup, Anja...
Molecular Genetics and Metabolism. 129 (2020) 2 - p. S78 , 2020

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

10

Efficacy and safety of arimoclomol in patients with Niemann..:

Patterson, Marc ; Mengel, Eugen ; Bembi, Bruno...
Molecular Genetics and Metabolism. 129 (2020) 2 - p. S127 , 2020

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

11

Metachromatic Leukodystrophy: An Approach to Treatment (P4...:

Krarup, Christian ; Dali, Christine i. ; Kraegeloh-Mann, Ingeborg..
Neurology. 92 (2019) 15_supplement - p. , 2019

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

12

Comprehensive long‐term efficacy and safety of recombinant ..:

Lund, Allan M. ; Borgwardt, Line ; Cattaneo, Federica...
Journal of Inherited Metabolic Disease. 41 (2018) 6 - p. 1225-1233 , 2018

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

13

Global treatment responder analysis demonstrates clinically..:

Harmatz, Paul ; Cattaneo, Federica ; Ardigò, Diego...
Molecular Genetics and Metabolism. 123 (2018) 2 - p. S58 , 2018

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

14

Efficacy and safety of Velmanase alfa in the treatment of p..:

Borgwardt, Line ; Guffon, Nathalie ; Amraoui, Yasmina...
Journal of Inherited Metabolic Disease. 41 (2018) 6 - p. 1215-1223 , 2018

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

15

Global treatment response analysis of velmanase alfa long t..:

Harmatz, Paul ; Cattaneo, Federica ; Ardigò, Diego...
Molecular Genetics and Metabolism. 123 (2018) 2 - p. S58 , 2018

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