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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 ...
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).
The Tubex system was widely used after World War II and expanded as a system of distributing and administration a large variety of drugs from antibiotics to vaccines in a pre-filled glass cartridge syringe with attached sterile needle. It aided in a standardization of an immediate use sterile dosage forms.
Gowers's sign is a medical sign that indicates weakness of the proximal muscles, namely those of the lower limb.The sign describes a patient that has to use their hands and arms to "walk" up their own body from a squatting position due to lack of hip and thigh muscle strength.
William Canfield is a glycobiologist, chief scientific officer and founder of an Oklahoma City-based biotechnology company, Novazyme, which was acquired by Genzyme in August 2001 and developed, among other things, an enzyme that can stabilize (but not cure) Pompe disease, based on Canfield's ongoing research since 1998.
A 54 mg tablet of Concerta, which uses OROS technology. 22% of the drug is contained in the red overcoat, while the remaining 78% is split between two drug layers of differing concentration. The tablet uses an additional push layer that expands as water enters the tablet via the osmotic membrane.
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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