Suchergebnisse - "Leukodystrophy"

PubPharm (1.977)

1

CSF1R-related disorder : State of the art, challenges, and proposition of a new terminology

enthalten in: Parkinsonism & related disorders | 2024

von Dulski, J. | Muthusamy, K. | Lund, T. | +1

2

RARS1-related hypomyelinating leukodystrophy-9 (HLD-9) in two distinct Iranian families : Case report and literature review

enthalten in: Molecular genetics & genomic medicine | 2024

von Biglari, S. | Vahidnezhad, H. | Tabatabaiefar, M. | +2

3

Imaging feature of Leukoencephalopathy with calcifications and cysts (Labrune syndrome)

enthalten in: World neurosurgery | 2024

von Gaikwad, S. | Charan, B. | Jain, S.

4

Spectrum of ERCC6-Related Cockayne Syndrome (Type B) : From Mild to Severe Forms

enthalten in: Genes | 2024

von Sartorelli, J. | Travaglini, L. | Macchiaiolo, M. | +15

5

Differential diagnosis between multiple sclerosis and leukodystrophies - A scoping review

enthalten in: Journal of the neurological sciences | 2024

von Chaer, L. | de Mendonça, J. | Del Negro, M. | +11

6

Loss of dihydroceramide desaturase drives neurodegeneration by disrupting endoplasmic reticulum and lipid droplet homeostasis in glial cells

enthalten in: bioRxiv : the preprint server for biology | 2024

von Zhu, Y. | Cho, K. | Lacin, H. | +5

7

Gene Transfer Clinical Trial for Krabbe Disease : A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE)

enthalten in: ClinicalTrials.gov | 2024

8

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT : A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)

enthalten in: ClinicalTrials.gov | 2024

9

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients : A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)

enthalten in: ClinicalTrials.gov | 2024

10

The 37TrillionCells initiative for improving global healthcare via cell-based interception and precision medicine : focus on neurodegenerative diseases

enthalten in: Molecular brain | 2024

von Coulombe, B. | Durcan, T. | Bernard, G. | +4

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