Catalog No.
AHC80001
Biological activity
Measured by its ability to release glucose from starch. The specific activity is >7,500 pmol/min/μg, as measured under the described conditions.
Expression system
Mammalian Cells
Species
Homo sapiens (Human)
Protein length
Ala70-Cys952
Nature
Recombinant
Endotoxin level
<0.1 EU/μg of the protein by the LAL method.
Purity
>90% as determined by SDS-PAGE.
Accession
P10253
Applications
Bioactivity, ELISA, Immunogen, SDS-PAGE, WB
Form
Lyophilized
Storage buffer
Lyophilized from a solution in PBS pH 7.4, 5% Trehalose, 5% Mannitol.
Reconstitution
Reconstitute in sterile water for a stock solution. A copy of datasheet will be provided with the products, please refer to it for details.
Shipping
In general, proteins are provided as lyophilized powder/frozen liquid. They are shipped out with dry ice/blue ice unless customers require otherwise.
Stability and Storage
Use a manual defrost freezer and avoid repeated freeze thaw cycles. Store at 2 to 8°C for one week. Store at -20 to -80°C for twelve months from the date of receipt.
Alternative Names
GAA, Lysosomal alpha-glucosidase, Acid maltase, Aglucosidase alfa
Improving the treatment of Pompe disease with enzyme replacement therapy: current strategies and clinical evidence., PMID:40237692
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
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
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
Greater Efficacy of Avalglucosidase vs Alglucosidase Alfa in Adult Pompe Disease? The Jury Is Still Out., PMID:35618439
Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease., PMID:35109913
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
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
White matter lesions in treated late onset Pompe disease are not different to matched controls., PMID:31153821
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease., PMID:31086307
Improved efficacy of a next-generation ERT in murine Pompe disease., PMID:30843882
Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa., PMID:30778879
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
Two successfully completed pregnancies in adult onset Pompe disease, under continued treatment with alglucosidase alfa., PMID:30715719
For research use only. Not for human or drug use.
