Detalhe da pesquisa
1.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788506
2.
Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.
Cytotherapy
; 2024 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38613540
3.
Transthoracic impedance variability to assess quality of chest compression in out-of-hospital cardiac arrest.
Acta Anaesthesiol Scand
; 68(4): 556-566, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38221650
4.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35065785
5.
Indoleamine 2,3-Dioxygenase Deletion to Modulate Kynurenine Pathway and to Prevent Brain Injury after Cardiac Arrest in Mice.
Anesthesiology
; 139(5): 628-645, 2023 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37487175
6.
Expanding the Spectrum of NUBPL-Related Leukodystrophy.
Neuropediatrics
; 54(3): 161-166, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36868263
7.
Characterization of the Cardiac Structure and Function of Conscious D2.B10-Dmdmdx/J (D2-mdx) mice from 16-17 to 24-25 Weeks of Age.
Int J Mol Sci
; 24(14)2023 Jul 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-37511564
8.
A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.
J Clin Immunol
; 42(8): 1742-1747, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35945378
9.
Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
J Pediatr
; 240: 297-301.e5, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34547335
10.
Searching for Preclinical Models of Acute Decompensated Heart Failure: a Concise Narrative Overview and a Novel Swine Model.
Cardiovasc Drugs Ther
; 36(4): 727-738, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33098053
11.
Cardiopulmonary Resuscitation-associated Lung Edema (CRALE). A Translational Study.
Am J Respir Crit Care Med
; 203(4): 447-457, 2021 02 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-32897758
12.
Propranolol Reduces the Development of Lesions and Rescues Barrier Function in Cerebral Cavernous Malformations: A Preclinical Study.
Stroke
; 52(4): 1418-1427, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33618555
13.
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
J Inherit Metab Dis
; 44(5): 1151-1164, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33855715
14.
Clinical practice recommendations on the management of perioperative cardiac arrest: A report from the PERIOPCA Consortium.
Crit Care
; 25(1): 265, 2021 07 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-34325723
15.
Efficacy of acute administration of inhaled argon on traumatic brain injury in mice.
Br J Anaesth
; 126(1): 256-264, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32977957
16.
Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry.
Anal Chem
; 92(9): 6341-6348, 2020 05 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31922725
17.
Kitcre knock-in mice fail to fate-map cardiac stem cells.
Nature
; 555(7697): E1-E5, 2018 03 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-29565363
18.
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Lancet
; 388(10043): 476-87, 2016 Jul 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27289174
19.
Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.
J Inherit Metab Dis
; 40(4): 543-554, 2017 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28560469
20.
Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy.
Hum Mutat
; 37(1): 16-27, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-26462614