Ads
related to: pombility medication for pompe 2 point system pdf
Search results
Results from the WOW.Com Content Network
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, 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.
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.
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.
Erythropoiesis-stimulating agents (ESA) are medications which stimulate the bone marrow to make red blood cells. [1] They are used to treat anemia due to end stage kidney disease, chemotherapy, major surgery, or certain treatments in HIV/AIDS. [1] [2] In these situations they decrease the need for blood transfusions. [2]
An infusion pump infuses fluids, medication or nutrients into a patient's circulatory system. It is generally used intravenously, although subcutaneous, arterial and epidural infusions are occasionally used. Infusion pumps can administer fluids in ways that would be impractically expensive or unreliable if performed manually by nursing staff.
Teduglutide, sold under the brand names Revestive (EU) and Gattex (US), is a 33-membered polypeptide and glucagon-like peptide-2 (GLP-2) analog that is used for the treatment of short bowel syndrome. It works by promoting mucosal growth and possibly restoring gastric emptying and secretion. [ 7 ]
Ads
related to: pombility medication for pompe 2 point system pdf