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/vufind/Search/Results?lookfor=%22Adang%2C+Laura%22&type=Person&sort=year
/vufind/Search/Results?lookfor=%22Adang%2C+Laura%22&type=Person&sort=year
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PubPharm (83)
1
Metachromatic leukodystrophy : A story of hope woven from sorrow
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2024
von
Adang, L.
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2
Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States
enthalten in:
Cytotherapy
| 2024
von
Adang, L.
|
Bonkowsky, J.
|
Boelens, J.
| +29
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3
Characterization of Fine Motor and Visual Motor Skills in Aicardi-Goutières Syndrome
enthalten in:
Journal of child neurology
| 2024
von
Cusack, S.
|
Gavazzi, F.
|
de Barcelos, I.
| +11
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4
Longitudinal natural history studies based on real-world data in rare diseases : Opportunity and a novel approach
enthalten in:
Molecular genetics and metabolism
| 2024
von
Adang, L.
|
Sevagamoorthy, A.
|
Sherbini, O.
| +20
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5
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
enthalten in:
bioRxiv.org
| 2024
von
Tricoli, L.
|
Sase, S.
|
Hacker, J.
| +16
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6
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of Multiple Sulfatase Deficiency
enthalten in:
bioRxiv.org
| 2024
von
Pham, V.
|
Tricoli, L.
|
Hong, X.
| +13
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7
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
enthalten in:
bioRxiv : the preprint server for biology
| 2024
von
Tricoli, L.
|
Sase, S.
|
Hacker, J.
| +16
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8
Biochemical signatures of disease severity in multiple sulfatase deficiency
enthalten in:
Journal of inherited metabolic disease
| 2024
von
Adang, L.
|
Mowafy, S.
|
Herbst, Z.
| +13
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9
Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management
enthalten in:
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
| 2024
von
Laugwitz, L.
|
Schoenmakers, D.
|
Adang, L.
| +25
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10
Nucleotide metabolism, leukodystrophies, and CNS pathology
enthalten in:
Journal of inherited metabolic disease
| 2024
von
Gavazzi, F.
|
Gonzalez, C.
|
Arnold, K.
| +7
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Molecular genetics and metabolism
5
Journal of child neurology
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Journal of inherited metabolic disease
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Orphanet journal of rare diseases
4
Journal of Inherited Metabolic Disease
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American journal of medical genetics. Part A
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56
Journal Article
29
Research Support, N.I.H., Extramural
24
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12
Leukodystrophy
8
leukodystrophy
7
Review
6
Case Reports
5
EC 1.8.-
5
EC 3.1.6.-
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Oxidoreductases Acting on Sulfur Group Donors
5
Rare diseases
5
Sulfatases
4
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4
Metachromatic leukodystrophy
4
SUMF1 protein, human
4
Systematic Review
3
11F24CG16M
3
Aicardi-Goutières syndrome
3
Care
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Erscheinungszeitraum
61
2020-
14
2010-2019
7
2000-2009
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Englisch
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