Cargando…

Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease

BACKGROUND: Enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA, alglucosidase alfa) has improved survival, motor outcomes, daily life activity and quality of life in Pompe patients. However, ERT in Pompe disease often induces formation of antibodies, which may reduce th...

Descripción completa

Detalles Bibliográficos
Autores principales:	van Kooten, Harmke A., Ditters, Imke A. M., Hoogeveen-Westerveld, Marianne, Jacobs, Edwin H., van den Hout, Johanna M. P., van Doorn, Pieter A., Pijnappel, W. W. M. Pim, van der Ploeg, Ans T., van der Beek, Nadine A. M. E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8812154/ https://www.ncbi.nlm.nih.gov/pubmed/35109913 http://dx.doi.org/10.1186/s13023-022-02175-2

Ejemplares similares

Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Extension of the Pompe mutation database by linking disease‐associated variants to clinical severity
por: Niño, Monica Y., et al.
Publicado: (2019)

Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients
por: Poelman, Esther, et al.
Publicado: (2020)

Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Enzymatic diagnosis of Pompe disease: lessons from 28 years of experience
por: Niño, Monica Y., et al.
Publicado: (2020)

Broad variation in phenotypes for common GAA genotypes in Pompe disease
por: Niño, Monica Y., et al.
Publicado: (2021)

The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease
por: Kuperus, Esther, et al.
Publicado: (2018)

Lysosomal glycogen accumulation in Pompe disease results in disturbed cytoplasmic glycogen metabolism
por: Canibano‐Fraile, Rodrigo, et al.
Publicado: (2022)

MRI changes in diaphragmatic motion and curvature in Pompe disease over time
por: Harlaar, Laurike, et al.
Publicado: (2022)

Chest MRI to diagnose early diaphragmatic weakness in Pompe disease
por: Harlaar, Laurike, et al.
Publicado: (2021)

Hearing in adults with Pompe disease
por: van der Beek, Nadine A. M. E., et al.
Publicado: (2011)

Effects of immunomodulation in classic infantile Pompe patients with high antibody titers
por: Poelman, E., et al.
Publicado: (2019)

Update of the Pompe variant database for the prediction of clinical phenotypes: Novel disease‐associated variants, common sequence variants, and results from newborn screening
por: de Faria, Douglas O. S., et al.
Publicado: (2020)

Diffusion tensor imaging of the brain in Pompe disease
por: van den Dorpel, Jan J. A., et al.
Publicado: (2022)

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
por: Liang, Qiushi, et al.
Publicado: (2022)

Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients
por: van der Meijden, Jan C., et al.
Publicado: (2018)

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease
por: van Gelder, Carin M., et al.
Publicado: (2014)

A genetic modifier of symptom onset in Pompe disease
por: Bergsma, Atze J., et al.
Publicado: (2019)

Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease
por: van der Wal, Erik, et al.
Publicado: (2017)

Lack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease
por: Schaaf, Gerben J., et al.
Publicado: (2015)

Is the brain involved in patients with late‐onset Pompe disease?
por: van den Dorpel, Jan J. A., et al.
Publicado: (2022)

Phenotypical variation within 22 families with Pompe disease
por: Wens, Stephan C A, et al.
Publicado: (2013)

From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides
por: Bergsma, Atze J, et al.
Publicado: (2016)

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
por: van Gelder, C. M., et al.
Publicado: (2016)

Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients
por: van den Dorpel, J. J. A., et al.
Publicado: (2020)

Effects of exercise training in 23 adults with Pompe disease receiving enzyme therapy
por: van den Berg, Linda, et al.
Publicado: (2013)

The quick motor function test: a new tool to rate clinical severity and motor function in Pompe patients
por: van Capelle, Carine I., et al.
Publicado: (2011)

Burden of illness of Pompe disease in patients only receiving supportive care
por: Kanters, Tim A., et al.
Publicado: (2011)

Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy
por: van der Meijden, Jan C., et al.
Publicado: (2018)

A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease
por: Bergsma, Atze J., et al.
Publicado: (2020)

Neurofilament Light and Its Association With CNS Involvement in Patients With Classic Infantile Pompe Disease
por: Mackenbach, Maarten J., et al.
Publicado: (2023)

Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease
por: Harlaar, Laurike, et al.
Publicado: (2019)

A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function
por: van Gelder, C, et al.
Publicado: (2013)

GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells
por: van der Wal, Erik, et al.
Publicado: (2017)

Minutes of the European POmpe Consortium (EPOC) Meeting March 27 to 28, 2015, Munich, Germany
por: Schoser, Benedikt, et al.
Publicado: (2015)

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy
por: Liang, Qiushi, et al.
Publicado: (2022)

Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease
por: Schaaf, Gerben J., et al.
Publicado: (2018)

Quantification of Diaphragm Mechanics in Pompe Disease Using Dynamic 3D MRI
por: Mogalle, Katja, et al.
Publicado: (2016)

Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study
por: Yuan, Meng, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_hv31np5g56i5jklioalaneu0mf