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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 is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. [14]In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).
Glucagon-like peptide-1 receptor agonists (GLP-1) drugs are a class of medications used to manage type 2 diabetes and treat obesity. While many drugs in this class are relatively new, researchers ...
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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 ...
February 2, 2025 at 1:30 AM A new opioid-free pain medication was approved by the U.S. Food and Drug Administration (FDA) on Thursday, marking a non-addictive alternative for patients.
Linagliptin, sold under the brand name Tradjenta among others, is a medication used to treat type 2 diabetes (but not type 1) in conjunction with exercise and diet. [8] [9] Linagliptin is a dipeptidyl peptidase-4 inhibitor [8] that works by increasing the production of insulin and decreasing the production of glucagon by the pancreas. [8]
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