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Avalglucosidase alfa, sold under the brand name Nexviazyme, is an enzyme replacement therapy medication used for the treatment of glycogen storage disease type II (Pompe disease). [ 6 ] [ 7 ]
Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [4] [5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase ...
In a study [23] which included the largest cohort of patients with Pompe disease treated with enzyme replacement therapy (ERT) to date findings showed that Myozyme treatment clearly prolongs ventilator-free survival and overall survival in patients with infantile-onset Pompe disease as compared to an untreated historical control population ...
The FDA has placed a clinical hold on Astellas Pharma Inc's (OTC: ALPMY) FORTIS Phase 1/2 trial following a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial ...
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Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). [6] Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha ...
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