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human GAA protein (Myozyme)

Subscribe to New Research on human GAA protein

Defects in the gene for this protein cause glycogen storage disease II, also known as Pompe disease, RefSeq NM_000152

Also Known As:

Myozyme; alglucosidase alfa; GAA protein, human; Genzyme brand of Recombinant human alglucosidase alfa; LYAG protein, human; acid alpha-glucosidase, human; acid maltase, human; glucosidase, alpha; acid (Pompe disease, glycogen storage disease type II), human; lysosomal alpha-glucosidase, human; recombinant human acid alpha-glucosidase; rhGAA

Networked: 96 relevant articles (23 outcomes, 20 trials/studies)

Relationship Network

Drug Context: Research Results

Enzymes and Coenzymes
- Enzymes: 34502
  - Hydrolases: 639
    - Glycoside Hydrolases: 275
      - Glucosidases: 578
        alpha-Glucosidases: 922
        human GAA protein: 96

Experts

1.	Kishnani, Priya S: 28 articles (11/2015 - 08/2005)
2.	Bali, Deeksha: 8 articles (01/2014 - 12/2008)
3.	Young, Sarah P: 8 articles (01/2014 - 08/2005)
4.	Thurberg, Beth L: 8 articles (02/2013 - 11/2004)
5.	Chen, Y T: 7 articles (11/2008 - 03/2001)
6.	Prater, Sean N: 6 articles (11/2015 - 08/2011)
7.	Case, Laura E: 6 articles (04/2015 - 05/2007)
8.	Banugaria, Suhrad G: 6 articles (02/2013 - 08/2011)
9.	Corzo, Deyanira: 6 articles (04/2010 - 10/2004)
10.	van der Ploeg, Ans T: 5 articles (04/2015 - 04/2010)

Related Diseases

1.	Glycogen Storage Disease Type II (Pompe's Disease) 08/01/2005 - "These results suggest that urinary Glc4 and plasma Hex4 could serve as a valuable adjunct to clinical endpoints for monitoring the efficacy of therapeutic interventions such as rhGAA ERT in Pompe disease." 04/01/2009 - "The results from clinical trials with recombinant human acid alpha-glucosidase have been published, and they show promising results with regards to the improvement of respiratory function in patients with Pompe disease. " 01/09/2007 - "Recombinant human acid alpha-glucosidase is safe and effective for treatment of infantile-onset Pompe disease. " 04/01/2015 - "This exploratory, randomized, open-label, 52-week study examined the safety and efficacy of 2 ERT regimens of alglucosidase alfa (20 mg/kg/week or 40 mg/kg/2 weeks) in 13 patients with Pompe disease and clinical decline or a lack of improvement on standard ERT: late-onset (n = 4), infantile-onset (n = 9). " 04/01/2015 - "Although treatment with alglucosidase alfa has helped improve the prognosis of patients with late-onset Pompe disease, both the development of the disease and the effectiveness of the treatment need to be monitored on a regular basis. "
2.	Cardiomyopathies (Cardiomyopathy) 01/01/2014 - "Enzyme replacement therapy using recombinant human GAA (rhGAA) has proven beneficial in addressing several aspects of the disease such as cardiomyopathy and aberrant motor function. " 08/01/2011 - "Enzyme replacement therapy with rhGAA (Myozyme®) has lead to improved survival, which is largely attributable to improvements in cardiomyopathy and skeletal muscle function. " 01/01/2005 - "Both clinical data and the results of preclinical studies in our knockout model of this disease show that rhGAA is much more effective in resolving the cardiomyopathy than the skeletal muscle myopathy. " 12/01/2010 - "Alglucosidase alpha (Myozyme(®)) improves the cardiomyopathy and life expectancy of infants suffering from Pompe disease and is under evaluation for the treatment of the juvenile and adult forms of the disease. " 07/29/2000 - "In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. "
3.	Muscular Diseases (Myopathy) 01/01/2005 - "Both clinical data and the results of preclinical studies in our knockout model of this disease show that rhGAA is much more effective in resolving the cardiomyopathy than the skeletal muscle myopathy. " 09/01/2011 - "Pompe disease (PD) is a progressive metabolic myopathy for which the only available treatment is alglucosidase alfa (Myozyme®). "
4.	Anaphylaxis (Anaphylactic Shock) 01/01/2014 - "Our studies indicate that lower enzyme doses reduce the antibody responses to rhGAA, reduce the incidence of immune toxicity and avoid ERT-associated anaphylaxis. " 11/01/2009 - "We report on the successful desensitization of an adult female with Pompe disease who had previously experienced anaphylaxis to intravenous alglucosidase alfa therapy. " 11/01/2009 - "Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa." 06/01/2012 - "This is further supported by IgG1 being the prevalent antibody subclass against rhGAA during ERT and consistent with prior reports on IgE formation and anaphylaxis in this model. "
5.	Hypersensitivity (Allergy) 10/01/2012 - "Animal and human studies of enzyme replacement therapy (ERT) for Pompe disease (PD) have indicated that antibodies (Abs) generated against infused recombinant human α-glucosidase (rhGAA) can have a negative impact on the therapeutic outcome and cause hypersensitivity reactions. " 12/01/2010 - "Over a period of 44 months, we monitored cognitive and motor development, behavior, auditory function, and brain imaging of a CRIM-negative infantile Pompe disease patient on rhGAA and monoclonal anti-immunoglobulin E (anti-IgE) antibody (omalizumab) treatment due to severe allergic reaction. "

Related Drugs and Biologics

1.	human GAA protein (Myozyme)
2.	alpha-Glucosidases (Acid Maltase)
3.	Glucosidases
4.	Antibodies
5.	Ligands
6.	Glycogen
7.	Mannose (D-Mannose)
8.	Immunoglobulins (Immunoglobulin)
9.	Immunoglobulin G (IgG)
10.	Immunoglobulin E (IgE)

Related Therapies and Procedures

1.	Enzyme Replacement Therapy
2.	Mechanical Ventilators (Ventilator)
3.	Enzyme Therapy
4.	Immunomodulation
5.	Drug-Eluting Stents