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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 safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion. [8] Myozyme treatment prolongs ventilator-free survival and overall survival. Early diagnosis and early treatment lead to much better outcomes.
Miglustat, sold under the brand name Zavesca among others, is a medication used to treat type I Gaucher disease [7] and Pompe disease. [10] It was approved for medical use in the European Union in November 2002, [7] [11] and for medical use in the United States in July 2003. [12] [13]
Most of these medications are benzimidazole derivatives, related to omeprazole, but imidazopyridine derivatives such as tenatoprazole have also been developed. [77] Potassium-competitive inhibitors such as revaprazan reversibly block the potassium-binding site of the proton pump, acting more quickly, but are not available in most countries.
Patient-controlled analgesia (PCA [1]) is any method of allowing a person in pain to administer their own pain relief. [2] The infusion is programmable by the prescriber. If it is programmed and functioning as intended, the machine is unlikely to deliver an overdose of medication. [3]
Brandon Hunter was arrested again Feb. 5 after he was caught with the drugs and in the same Midtown spot, according to records and sources. NYC’s ‘worst drug dealer,’ 20, busted again with ...
Antipsychotics by class Generic name Brand names Chemical class ATC code Typical antipsychotics; Acepromazine: Atravet, Acezine: phenothiazine: N05AA04
Ramona Bates MD, writing for the health news organisation, EmaxHealth, stated that the film brings attention to Pompe disease. [18] Peter Rainer from The Christian Science Monitor mentions that Big Pharma got a surprisingly free pass in the film and that it will come as a surprise to all those sufferers struggling to get orphan drugs developed ...
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