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Also known as recombinant human acid alpha-glucosidase (rhGAA)
recombinant human acid alpha-glucosidase · 1 trial · 4 indications
| Name | Type | Description |
|---|---|---|
| recombinant human acid alpha-glucosidase (rhGAA) | DRUG | - |
Inclusion Criteria: * Clinical diagnosis of Classical Infantile Pompe Disease * endogenous GAA activity \< 1.0% * cardiomegaly * cardiomyopathy * CRIM (+) * ability to comply with the clinical protocol which will require extensive clinical evaluations Exclusion Criteria: * respiratory insufficien...
Recombinant human acid alpha-glucosidase is used for the treatment of Pompe disease, a rare inherited glycogen storage disorder also known as glycogen storage disease type II or acid maltase deficiency. It is being developed by Sanofi as an enzyme replacement approach for patients with this condition.
Recombinant human acid alpha-glucosidase is an exogenous protein designed to target F9. It is intended to supply a functional form of the acid alpha-glucosidase enzyme, which is deficient in Pompe disease. By providing this enzyme, the therapy aims to address the underlying enzyme deficiency that leads to glycogen accumulation.
Sanofi is developing recombinant human acid alpha-glucosidase. Sanofi trades on the New York Stock Exchange under the ticker SNY. The company is responsible for the clinical development of this enzyme replacement therapy for Pompe disease.
Recombinant human acid alpha-glucosidase is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 2 trial for this drug has been completed.
Recombinant human acid alpha-glucosidase has been studied in one completed Phase 2 clinical trial, NCT00025896, titled Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease. The trial enrolled 8 participants in the United States and was an uncontrolled, non-randomized study.
Yes, recombinant human acid alpha-glucosidase is also known as rhGAA. The abbreviation rhGAA stands for recombinant human acid alpha-glucosidase, which is the full name of the therapy being developed by Sanofi for Pompe disease.