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1.
Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy.
Mol Genet Metab
; 123(2): 85-91, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-29050825
2.
CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.
Genet Med
; 17(11): 912-8, 2015 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-25741864
3.
Structures of the compact helical core domains of feline calicivirus and murine norovirus VPg proteins.
J Virol
; 87(10): 5318-30, 2013 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-23487472
4.
Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease.
Genet Med
; 15(2): 123-31, 2013 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-23060045
5.
The emerging phenotype of long-term survivors with infantile Pompe disease.
Genet Med
; 14(9): 800-10, 2012 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-22538254
6.
Autopsy findings in late-onset Pompe disease: a case report and systematic review of the literature.
Mol Genet Metab
; 106(4): 462-9, 2012 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-22664150
7.
The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.
Genet Med
; 13(8): 729-36, 2011 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-21637107
8.
Atypical immunologic response in a patient with CRIM-negative Pompe disease.
Mol Genet Metab
; 104(4): 583-6, 2011 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-21889385
9.
CORRIGENDUM: CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.
Genet Med
; 17(7): 596, 2015 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-26133565
10.
Letter to the Editors: Concerning "CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy" by Al Khallaf et al.
J Inherit Metab Dis
; 37(1): 141-3, 2014 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-23887636
11.
Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses.
JCI Insight
; 1(11)2016 Jul 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-27493997
12.
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.
Mol Genet Metab Rep
; 1: 446-450, 2014.
Artículo
en Inglés
| MEDLINE | ID: mdl-27896120
13.
Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.
Orphanet J Rare Dis
; 8: 90, 2013 Jun 20.
Artículo
en Inglés
| MEDLINE | ID: mdl-23787031
14.
Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT.
PLoS One
; 8(6): e67052, 2013.
Artículo
en Inglés
| MEDLINE | ID: mdl-23825616
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