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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 ...
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 ]
In 2004, he became a director at Amicus Therapeutics, based in Cranbury, New Jersey, and in January 2005, he was named the president and CEO of the company. [17] Amicus works to develop treatments for rare, devastating genetic disorders. The company has a Pompe treatment in development, as well as Galafold , which is for Fabry disease.
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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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 ...
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 ...
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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