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1.
Phase 3 Trial of Interleukin-1 Trap Rilonacept in Recurrent Pericarditis.
N Engl J Med
; 384(1): 31-41, 2021 01 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-33200890
2.
A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy.
Genet Med
; 16(6): 435-41, 2014 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-24202085
3.
Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: the Transthyretin Amyloidosis Cardiac Study (TRACS).
Am Heart J
; 164(2): 222-228.e1, 2012 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-22877808
4.
GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN).
J Cyst Fibros
; 19(2): 292-298, 2020 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-31594690
5.
An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy.
Drug Des Devel Ther
; 10: 1771-81, 2016.
Artículo
en Inglés
| MEDLINE | ID: mdl-27307708
6.
Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).
Orphanet J Rare Dis
; 10: 50, 2015 Apr 24.
Artículo
en Inglés
| MEDLINE | ID: mdl-25902842
7.
Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment.
Mol Genet Metab Rep
; 5: 103-106, 2015 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-28649553
8.
A Cerebrospinal Fluid Collection Study in Pediatric and Adult Patients With Hunter Syndrome
J. inborn errors metab. screen
; 3: e150002, 2015. tab, graf
Artículo
en Inglés
|
LILACS-Express
| ID: biblio-1090864
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