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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 ...
These trials enrolled 124 participants with late-onset Pompe disease and 22 participants with infantile-onset Pompe disease. [9] The participants were from 22 countries around the world, including the United States. [9] Avalglucosidase alfa was evaluated in four trials of 146 participants with Pompe disease. [9]
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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 ...
The treatment appears to be successful. One doctor said the patient, now 16 months old, looks like “any typically developing baby at this age.” With help from Duke doctors, a rare genetic ...
SPK-3006 is an experimental gene therapy developed for Pompe disease by Spark Therapeutics. It is delivered via adeno-associated virus and is intended to increase alpha-glucosidase production in the liver. [1] [2]
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