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1.
Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program.
Mol Genet Metab
; 136(2): 152-162, 2022 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-35459555
2.
Impact of newborn screening on the reported incidence and clinical outcomes associated with medium- and long-chain fatty acid oxidation disorders.
Genet Med
; 23(5): 816-829, 2021 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-33495527
3.
Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10-year pharmacovigilance analysis.
Br J Haematol
; 185(2): 297-310, 2019 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-30768680
4.
Eculizumab reduces complement activation, inflammation, endothelial damage, thrombosis, and renal injury markers in aHUS.
Blood
; 125(21): 3253-62, 2015 May 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-25833956
5.
Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome.
Kidney Int
; 89(3): 701-11, 2016 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-26880462
6.
Terminal Complement Inhibitor Eculizumab in Adult Patients With Atypical Hemolytic Uremic Syndrome: A Single-Arm, Open-Label Trial.
Am J Kidney Dis
; 68(1): 84-93, 2016 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-27012908
7.
Efficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studies.
Kidney Int
; 87(5): 1061-73, 2015 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-25651368
8.
Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria.
Pediatr Blood Cancer
; 61(9): 1544-50, 2014 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-24777716
9.
Assessment of human antihuman antibodies to eculizumab after long-term treatment in patients with paroxysmal nocturnal hemoglobinuria.
Am J Hematol
; 91(3): E16-7, 2016 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-26690023
10.
Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy.
J Neuromuscul Dis
; 8(2): 225-234, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-33459658
11.
A phase 2 clinical trial of deforolimus (AP23573, MK-8669), a novel mammalian target of rapamycin inhibitor, in patients with relapsed or refractory hematologic malignancies.
Clin Cancer Res
; 14(9): 2756-62, 2008 May 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-18451242
12.
Phase I study of samalizumab in chronic lymphocytic leukemia and multiple myeloma: blockade of the immune checkpoint CD200.
J Immunother Cancer
; 7(1): 227, 2019 08 23.
Artículo
en Inglés
| MEDLINE | ID: mdl-31443741
13.
Clinical trial data available for UX001, aceneuramic acid extended-release.
J Neurol Sci
; 411: 116724, 2020 04 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-32058180
14.
Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial.
Int J Hematol
; 98(4): 406-16, 2013 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-23934275
15.
Analysis of the pharmacodynamic activity of the mTOR inhibitor ridaforolimus (AP23573, MK-8669) in a phase 1 clinical trial.
Cancer Chemother Pharmacol
; 69(5): 1369-77, 2012 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-22231376
16.
Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial.
Int J Hematol
; 93(1): 36-46, 2011 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-21222185
17.
Phase I trial of the novel mammalian target of rapamycin inhibitor deforolimus (AP23573; MK-8669) administered intravenously daily for 5 days every 2 weeks to patients with advanced malignancies.
J Clin Oncol
; 26(3): 361-7, 2008 Jan 20.
Artículo
en Inglés
| MEDLINE | ID: mdl-18202410
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