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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.
GLP-1 and SGLT-2 drugs may be the best treatments for people with type 2 diabetes and COPD. Image credit: Visual Vic/Getty Images. This article originally appeared on Medical News Today
This is the first class of non-opioid pain medication approved to treat moderate to severe acute pain approved by the FDA in more than 20 years. FDA approves new type of non-opioid pain medication ...
The medication will be sold under the brand name Journavx for $15.50 per pill, according to Vertex Pharmaceuticals, the company that developed the new drug.
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.
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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