| 词条 | Elosulfase alfa |
| 释义 |
| drug_name = | IUPAC_name = | image = | alt = | caption = | tradename = Vimizim | Drugs.com = {{Drugs.com|MTM|elosulfase-alfa}} | MedlinePlus = | pregnancy_AU = | pregnancy_US = C | pregnancy_category= | licence_EU = yes | legal_AU = | legal_CA = | legal_UK = | legal_US = Rx-only | legal_status = | routes_of_administration = | bioavailability = | protein_bound = | metabolism = | elimination_half-life = | excretion = | CAS_number = 9025-60-9 | ATCvet = | ATC_prefix = A16 | ATC_suffix = AB12 | PubChem = | IUPHAR_ligand = 7392 | DrugBank = | ChemSpiderID = none | KEGG = D10333 | C=5020 | H=7588 | N=1364 | O=1418 | S=34 | molecular_weight = 110.8 kg/mol }} Elosulfase alfa (trade name Vimizim) is a drug for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase. Elosulfase alfa is a synthetic version of this enzyme. Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and approved for use in the US by the Food and Drug Administration in 2014.[1] The drug is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A.[2] Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper.[3] The cost of elosulfase alfa in some countries is $400,000 a year, which has made it difficult for some health systems to afford it.[4] References1. ^{{cite web | url = http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm386008.htm | title = FDA approves Vimizim to treat rare congenital enzyme disorder | publisher = Food and Drug Administration | date = Feb 14, 2014}} {{gastrointestinal-drug-stub}}2. ^{{cite journal |author= Hendriksz, C. et. al|title=Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study|journal=Journal of Inherited Metabolic Disease |volume=37 |issue=6|pages=979–990|date=November 2014|doi=10.1007/s10545-014-9715-6|pmid=24810369|pmc=4206772}} 3. ^{{cite journal |author= Hendriksz, C. et. al|title=Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial|journal=Molecular Genetics and Metabolism |volume=114 |issue=2|pages=178–185|date=February 2015|doi=10.1016/j.ymgme.2014.08.012 |pmid=25284089}} 4. ^Providing $400,000 life-saving drug to Australian kids with rare disease. The Australian Government will provide a life-saving treatment to Australian patients who have a rare medical condition known as Morquio A Syndrome, at no cost. The Hon Greg Hunt MP. Minister for Health. 15 June 2017 1 : Peptides |
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