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1.
A rare homozygous missense GDF2 (BMP9) mutation causing PAH in siblings: Does BMP10 status contribute?
Am J Med Genet A
; 191(1): 228-233, 2023 Jan.
Article
in English
| MEDLINE | ID: mdl-36259599
2.
At three years, patients with acute lymphoblastic leukaemia are still at risk for relapse. Results of the international MRC UKALLXII/ECOG E2993 trial.
Br J Haematol
; 191(1): 37-43, 2020 10.
Article
in English
| MEDLINE | ID: mdl-32220069
3.
An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.
Genet Med
; 21(4): 887-895, 2019 04.
Article
in English
| MEDLINE | ID: mdl-30214072
4.
Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial.
Lancet
; 390(10089): 50-61, 2017 07 01.
Article
in English
| MEDLINE | ID: mdl-28526489
5.
Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency).
Genet Med
; 18(1): 34-40, 2016 Jan.
Article
in English
| MEDLINE | ID: mdl-25834946
6.
Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies.
Mol Genet Metab
; 117(4): 419-26, 2016 Apr.
Article
in English
| MEDLINE | ID: mdl-26920513
7.
Transient low-dose methotrexate generates B regulatory cells that mediate antigen-specific tolerance to alglucosidase alfa.
J Immunol
; 193(8): 3947-58, 2014 Oct 15.
Article
in English
| MEDLINE | ID: mdl-25210119
8.
Population deworming every 6 months with albendazole in 1 million pre-school children in North India: DEVTA, a cluster-randomised trial.
Lancet
; 381(9876): 1478-86, 2013 Apr 27.
Article
in English
| MEDLINE | ID: mdl-23498850
9.
Transient low-dose methotrexate induces tolerance to murine anti-thymocyte globulin and together they promote long-term allograft survival.
J Immunol
; 189(2): 732-43, 2012 Jul 15.
Article
in English
| MEDLINE | ID: mdl-22711884
10.
2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV TAb Post-Dose Assessment, NanoString Validation, ELISpot as Gold Standard (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Biotherapeutics Immunogenicity & Risk Assessment; ADA/NAb Assay/Reporting Harmonization).
Bioanalysis
; 16(7): 77-119, 2024 Apr.
Article
in English
| MEDLINE | ID: mdl-38389403
11.
Thiopurine methyltransferase genotype-phenotype discordance and thiopurine active metabolite formation in childhood acute lymphoblastic leukaemia.
Br J Clin Pharmacol
; 76(1): 125-36, 2013 Jul.
Article
in English
| MEDLINE | ID: mdl-23252716
12.
A North American, single-center experience implanting fenestrated atrial devices and atrial flow regulators into a heterogeneous group of pediatric pulmonary hypertension patients.
Front Pediatr
; 11: 1073336, 2023.
Article
in English
| MEDLINE | ID: mdl-36762281
13.
Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study.
Clin Pharmacol Drug Dev
; 12(12): 1185-1193, 2023 12.
Article
in English
| MEDLINE | ID: mdl-37705424
14.
Multicenter review of a tadalafil suspension formulation for infants and children with pulmonary hypertension: A North American experience.
Front Pediatr
; 11: 1055131, 2023.
Article
in English
| MEDLINE | ID: mdl-36776903
15.
A Canadian, retrospective, multicenter experience with selexipag for a heterogeneous group of pediatric pulmonary hypertension patients.
Front Pediatr
; 11: 1055158, 2023.
Article
in English
| MEDLINE | ID: mdl-36925667
16.
Failure to tolerate continuous subcutaneous treprostinil in pediatric pulmonary hypertension patients.
Pulm Circ
; 13(2): e12224, 2023 Apr.
Article
in English
| MEDLINE | ID: mdl-37143765
17.
Monitoring and reporting the US COVID-19 vaccination effort.
Vaccine
; 2023 Dec 13.
Article
in English
| MEDLINE | ID: mdl-38097453
18.
Cathelicidin antimicrobial peptide expression is not induced or required for bacterial clearance during salmonella enterica infection of human monocyte-derived macrophages.
Infect Immun
; 80(11): 3930-8, 2012 Nov.
Article
in English
| MEDLINE | ID: mdl-22927052
19.
Outcomes in older adults with acute lymphoblastic leukaemia (ALL): results from the international MRC UKALL XII/ECOG2993 trial.
Br J Haematol
; 157(4): 463-71, 2012 May.
Article
in English
| MEDLINE | ID: mdl-22409379
20.
Response to de Vries et al.
Genet Med
; 19(11): 1281-1282, 2017 11.
Article
in English
| MEDLINE | ID: mdl-28492534