E-LIB Suche - Ergebnisse für: Sevin, Caroline

1

Consensus guidelines for the monitoring and management of m..:

Adang, Laura A. ; Bonkowsky, Joshua L. ; Boelens, Jaap Jan...
Cytotherapy. 26 (2024) 7 - p. 739-748 , 2024

2

Cell-based device provides effective therapeutic strategy t..:

Audouard, Emilie ; Banchi, Elena Gaia ; Alonso, Rafael...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107759 , 2024

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

3

Improving paediatric movement disorders care: Insights on r..:

Amato, María Eugenia ; Darling, Alejandra ; Stovickova, Lucie...
European Journal of Paediatric Neurology. , 2024

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

4

A phase 2 study assessing TAK-611150 mg intrathecal weekly ..:

Adang, Laura A. ; Malanga, C.J. ; Burton, Barbara K....
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107744 , 2024

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

5

Dose-response evaluation of intravenous gene therapy in a s..:

Audouard, Emilie ; Khefif, Nicolas ; Mansat, Charlotte...
Molecular Therapy - Methods & Clinical Development. 32 (2024) 2 - p. 101248 , 2024

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

6

Inventory of current practices regarding hematopoietic stem..:

Schoenmakers, Daphne H. ; Mochel, Fanny ; Adang, Laura A....
Orphanet Journal of Rare Diseases. 19 (2024) 1 - p. , 2024

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

7

Intravenous gene therapy using AAVPHP.eB for metachromatic ..:

Audouard, Emilie ; Banchi, Elena Gaia ; Alonso, Rafael...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107758 , 2024

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

8

Newborn screening in metachromatic leukodystrophy – Europea..:

Laugwitz, Lucia ; Schoenmakers, Daphne H. ; Adang, Laura A....
European Journal of Paediatric Neurology. 49 (2024) - p. 141-154 , 2024

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

9

Atidarsagene Autotemcel, a European Post-Regulatory Approva..:

Wynn, Robert F. ; Jones, Simon ; Fumagalli, Francesca...
Transplantation and Cellular Therapy. 29 (2023) 2 - p. S227 , 2023

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

10

Atidarsagene autotemcel, a European post-regulatory approva..:

Jones, Simon ; Wynn, Robert ; Fumagalli, Francesca...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107172 , 2023

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

11

Effects of miglustat therapy on neurological disorder and s..:

Freihuber, Cécile ; Dahmani-Rabehi, Bahia ; Brassier, Anaïs...
Orphanet Journal of Rare Diseases. 18 (2023) 1 - p. , 2023

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

12

A Retrospective Multicentric Study of 34 Patients with Niem..:

Gardin, Antoine ; Mussini, Charlotte ; Héron, Bénédicte...
The Journal of Pediatrics. 254 (2023) - p. 75-82.e4 , 2023

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

13

Childhood cerebral adrenoleukodystrophy (CCALD) in France: ..:

Sevin, Caroline ; Hatteb, Samira ; Clément, Aurore...
Orphanet Journal of Rare Diseases. 18 (2023) 1 - p. , 2023

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

14

Gene therapy for neurodegenerative disorders in children: d..:

Boespflug-Tanguy, Odile ; Sevin, Caroline ; Piguet, Francoise
Archives de Pédiatrie. 30 (2023) 8 - p. 8S32-8S40 , 2023

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

15

Prospective Multicenter Validation of a Simple Blood Test f..:

Mochel, Fanny ; Gras, Domitille ; Luton, Marie-Pierre...
Neurology. 100 (2023) 23 - p. , 2023

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