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.
about
Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responsesB-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case StudyEnhanced efficacy from gene therapy in Pompe disease using coreceptor blockadePompe disease: literature review and case seriesEnzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.Toward reducing immunogenicity of enzyme replacement therapy: altering the specificity of human β-glucuronidase to compensate for α-iduronidase deficiency.Innate and Adaptive Immune Response in Fabry DiseaseEnzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vectorCRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapySynergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.Treatment of lysosomal storage disorders: successes and challenges.Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy.Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.The Outcome of Infantile Onset Pompe Disease in South of Iran.Letter to the Editors: Concerning "Divergent clinical outcomes of alphaglucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state" by Takashi M et alCurrent and Future Treatments for Lysosomal Storage Disorders.A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I.Immunomodulatory, liver depot gene therapy for Pompe disease.Cardiac response to enzyme replacement therapy in infantile Pompe disease with severe hypertrophic cardiomyopathy.
P2860
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P2860
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.
description
2013 nî lūn-bûn
@nan
2013 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
name
Algorithm for the early diagno ...... improving the efficacy of ERT.
@ast
Algorithm for the early diagno ...... improving the efficacy of ERT.
@en
Algorithm for the early diagno ...... improving the efficacy of ERT.
@nl
type
label
Algorithm for the early diagno ...... improving the efficacy of ERT.
@ast
Algorithm for the early diagno ...... improving the efficacy of ERT.
@en
Algorithm for the early diagno ...... improving the efficacy of ERT.
@nl
prefLabel
Algorithm for the early diagno ...... improving the efficacy of ERT.
@ast
Algorithm for the early diagno ...... improving the efficacy of ERT.
@en
Algorithm for the early diagno ...... improving the efficacy of ERT.
@nl
P2093
P2860
P1433
P1476
Algorithm for the early diagno ...... improving the efficacy of ERT.
@en
P2093
Amy S Rosenberg
Catherine W Rehder
Christie Milleson
Deeksha S Bali
Francois Labarthe
Joel Charrow
Julian A J Raiman
Kathryn B Sheets
Paul Harmatz
Pranesh Chakraborty
P2860
P304
P356
10.1371/JOURNAL.PONE.0067052
P407
P577
2013-06-25T00:00:00Z