Alglucosidase

Clinical and genetic characteristics of infantile-onset Pompe disease and long-term efficacy of enzyme replacement therapy

Journal: Zhonghua er ke za zhi = Chinese journal of pediatrics

Published: May 14, 2026

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.

Journal: Journal of comparative effectiveness research

Published: February 27, 2026

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Journal: Health technology assessment (Winchester, England)

Published: February 16, 2026

Early enzyme replacement therapy in late-onset Pompe disease diagnosed by newborn screening.

Journal: Molecular genetics and metabolism

Published: January 25, 2026

Response to: Correspondence on 'Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements' by Barzel et al.

Journal: European journal of hospital pharmacy : science and practice

Published: December 31, 2025

Correspondence on "Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements" by Barzel et al.

Journal: European journal of hospital pharmacy : science and practice

Published: December 23, 2025

Short-Term Intensive Avalglucosidase Alfa Regimen in Late-Diagnosed Infantile Pompe Disease: A Case Report.

Journal: Reports (MDPI)

Published: December 12, 2025

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.

Journal: Frontiers in neurology

Published: November 29, 2025

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease.

Journal: Journal of neurology

Published: November 19, 2025

Alglucosidase alfa demonstrates effectiveness and safety in Chinese patients with late-onset Pompe disease: A multi-center prospective study.

Journal: Molecular genetics and metabolism

Published: November 11, 2025

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy

Published: November 09, 2025

Combined omalizumab and desensitization to control IgE-mediated hypersensitivity in enzyme replacement therapy for late-onset Pompe disease.

Journal: Orphanet journal of rare diseases

Published: October 23, 2025

Clinical and genetic characteristics of infantile-onset Pompe disease​ and long-term efficacy of enzyme replacement therapy

Clinical and genetic characteristics of infantile-onset Pompe disease​ and long-term efficacy of enzyme replacement therapy

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Early enzyme replacement therapy in late-onset Pompe disease diagnosed by newborn screening.

Early enzyme replacement therapy in late-onset Pompe disease diagnosed by newborn screening.

Response to: Correspondence on 'Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements' by Barzel et al.

Response to: Correspondence on 'Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements' by Barzel et al.

Correspondence on "Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements" by Barzel et al.

Correspondence on "Stability of alglucosidase alfa in 0.9% sodium chloride for enzyme replacement therapy in patients with Pompe disease: insights from enzyme activity and cellular uptake measurements" by Barzel et al.

Short-Term Intensive Avalglucosidase Alfa Regimen in Late-Diagnosed Infantile Pompe Disease: A Case Report.

Short-Term Intensive Avalglucosidase Alfa Regimen in Late-Diagnosed Infantile Pompe Disease: A Case Report.

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease.

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease.

Alglucosidase alfa demonstrates effectiveness and safety in Chinese patients with late-onset Pompe disease: A multi-center prospective study.

Alglucosidase alfa demonstrates effectiveness and safety in Chinese patients with late-onset Pompe disease: A multi-center prospective study.

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?

Combined omalizumab and desensitization to control IgE-mediated hypersensitivity in enzyme replacement therapy for late-onset Pompe disease.

Combined omalizumab and desensitization to control IgE-mediated hypersensitivity in enzyme replacement therapy for late-onset Pompe disease.

Clinical and genetic characteristics of infantile-onset Pompe disease and long-term efficacy of enzyme replacement therapy

Clinical and genetic characteristics of infantile-onset Pompe disease and long-term efficacy of enzyme replacement therapy