Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis, PMID: 27372449
A randomized study of alglucosidase alfa in late-onset Pompe's disease, PMID: 20393176
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study, PMID: 30770310
Alglucosidase Alfa, PMID: 30000880
Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature, PMID: 31904026
The emerging phenotype of late-onset Pompe disease: A systematic literature review, PMID: 28185884
Gene Therapy for Pompe Disease: The Time is now, PMID: 31298581
Pre/post effectiveness evaluation of updated additional risk minimisation measures for an orphan disease: Myozyme (alglucosidase alfa) Safety Information Packet, PMID: 31667955
Pompe disease: early diagnosis and early treatment make a difference, PMID: 23632029
Structure of human lysosomal acid α-glucosidase-a guide for the treatment of Pompe disease, PMID: 29061980
Recent developments, utilization, and spending trends for pompe disease therapies, PMID: 24991319
Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease, PMID: 28648663
Alglucosidase alfa and Pompe disease: still going strong?, PMID: 23034445
Guidance for the care of neuromuscular patients during the COVID-19 pandemic outbreak from the French Rare Health Care for Neuromuscular Diseases Network, PMID: 32354651
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease, PMID: 19287243
Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry, PMID: 32515844
Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients, PMID: 32623214
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa, PMID: 23031366
Alglucosidase alfa therapy for Pompe disease in pregnancy - Case report, PMID: 28320122
Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial, PMID: 19649685
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease, PMID: 29237491
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, PMID: 26768149
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study, PMID: 29565424
An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions, PMID: 21802969
Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease, PMID: 25617983
Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for a patient presenting with a PRKAG2 mutation, PMID: 27692944
Identification of Undetected Monogenic Cardiovascular Disorders, PMID: 32792077
High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa, PMID: 20826098
Improved efficacy of a next-generation ERT in murine Pompe disease, PMID: 30843882
Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III, PMID: 23318145
Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa, PMID: 19640753
Glycogen storage disease types I and II: treatment updates, PMID: 17308886
Acid alpha-glucosidase deficiency (Pompe disease), PMID: 17217857
Lysosomal storage diseases, PMID: 18720909
Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study, PMID: 27473031
Therapeutic approaches in glycogen storage disease type II/Pompe Disease, PMID: 19019308
Two successfully completed pregnancies in adult onset Pompe disease, under continued treatment with alglucosidase alfa, PMID: 30715719
Transient low-dose methotrexate generates B regulatory cells that mediate antigen-specific tolerance to alglucosidase alfa, PMID: 25210119
Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation, PMID: 29289479
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease, PMID: 19542901
Immunomodulatory, liver depot gene therapy for Pompe disease, PMID: 29295737
[Which follow-up for innovative treatments?], PMID: 30943166
Feasibility Study for Bedside Production of Recombinant Human Acid α-Glucosidase: Technical and Financial Considerations, PMID: 32065100
[Pompe's disease], PMID: 18029059
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease, PMID: 24884717
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy, PMID: 22290025
Massive parallel sequencing of dried umbilical cord remnants, PMID: 32902111
Diagnosis and management of Pompe disease, PMID: 25118547
Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy, PMID: 30314719
FDA balks at Myozyme scale-up, PMID: 18536660
Improving the treatment of Pompe disease with enzyme replacement therapy: current strategies and clinical evidence., PMID:40237692
Efficacy of transitioning from alglucosidase alfa to avalglucosidase alfa in infantile-onset Pompe disease: A single-center cohort analysis., PMID:39927452
A Real-World Data Analysis of Alglucosidase Alfa in the FDA Adverse Event Reporting System (FAERS) Database., PMID:39833603
Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease., PMID:39775064
Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease., PMID:39632463
[Importance of early treatment and quantitative evaluation of enzyme replacement therapy for Pompe disease: alglucosidase alfa post-marketing surveillance additional analysis]., PMID:39566964
Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials., PMID:39566417
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease., PMID:39535661
Pompe disease: Unmet needs and emerging therapies., PMID:39418752
Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data., PMID:39287071
Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease., PMID:39154400
Long-term observation of patients with advanced late-onset Pompe disease undergoing enzyme replacement therapy: A 15-year observation in a single center., PMID:39142946
Real-world data of in-hospital administration of alglucosidase alfa in French patients with Pompe disease: results from the National Claims Database., PMID:38963441
Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry., PMID:38896264
Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease., PMID:38768612
Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy., PMID:38715621
Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study., PMID:38587143
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)., PMID:38418563
Effect of intracerebroventricular administration of alglucosidase alfa in two mouse models of Lafora disease: Relevance for clinical practice., PMID:38341935
Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa., PMID:38313679
Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial., PMID:38184428
Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa., PMID:38160363
Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry., PMID:38057861
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)., PMID:38057636
Safety outcomes and patients' preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond., PMID:37891668
Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease., PMID:37765007
Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review., PMID:37759814
Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study., PMID:37705424
Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice., PMID:37679046
Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions., PMID:37326923
Model-Informed Approach Supporting Approval of Nexviazyme (Avalglucosidase Alfa-ngpt) in Pediatric Patients with Late-Onset Pompe Disease., PMID:36653728
Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease., PMID:36623499
1,6-epi-Cyclophellitol Cyclosulfamidate Is a Bona Fide Lysosomal α-Glucosidase Stabilizer for the Treatment of Pompe Disease., PMID:35917590
Greater Efficacy of Avalglucosidase vs Alglucosidase Alfa in Adult Pompe Disease? The Jury Is Still Out., PMID:35618439
Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium., PMID:34822769
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial., PMID:34800400
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial., PMID:34800399
Avalglucosidase alfa: First Approval., PMID:34591286
First successful concomitant therapy of immune tolerance induction therapy and desensitization in a CRIM-negative infantile Pompe patient., PMID:34561975
Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease., PMID:34220802
STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa., PMID:33543425
Expansion of immature, nucleated red blood cells by transient low-dose methotrexate immune tolerance induction in mice., PMID:33205401
Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort., PMID:32849613
Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients., PMID:32623214
Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry., PMID:32515844
Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease., PMID:32290314
Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature., PMID:31904026
Pre/post effectiveness evaluation of updated additional risk minimisation measures for an orphan disease: Myozyme (alglucosidase alfa) Safety Information Packet., PMID:31667955
For research use only. Not for human or drug use.
