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Elosulfase Alfa
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. The drug is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper. The cost of elosulfase alfa in some countries is $2,080,000-$6,240,000 a year, which has made it difficult for some health systems to afford it. In June 2019, a Belgian court issued a preliminary injunction forcing BioMarin to continue supplying Vimizim to a young girl suffering from Morquio syndrome free of charge. BioMarin stopped providing the dr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Morquio Syndrome
Morquio syndrome, also known as Mucopolysaccharidosis Type IV (MPS IV), is a rare metabolic disorder in which the body cannot process certain types of sugar molecules called glycosaminoglycans (AKA GAGs, or mucopolysaccharides). In Morquio syndrome, the specific GAG which builds up in the body is called keratan sulfate. This birth defect, which is autosomal recessive, is a type of lysosomal storage disorder. The buildup of GAGs in different parts of the body causes symptoms in many different organ systems. In the US, the incidence rate for Morquio syndrome is estimated at between 1 in 200,000 and 1 in 300,000 live births. Signs and symptoms Patients with Morquio syndrome appear healthy at birth. Types A and B have similar presentations, but Type B generally has milder symptoms. The age of onset is usually between 1 and 3 years of age. Morquio syndrome causes progressive changes to the skeleton of the ribs and chest, which may lead to neurological complications such as nerve ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
N-acetylgalactosamine-6-sulfatase
The enzyme ''N''-acetylgalactosamine-6-sulfatase (EC 3.1.6.4) catalyzes the chemical reaction of cleaving off the 6-sulfate groups of the ''N''-acetyl-D-galactosamine 6-sulfate units of the macromolecule chondroitin sulfate and, similarly, of the D-galactose 6-sulfate units of the macromolecule keratan sulfate. This enzyme belongs to the family of hydrolases, specifically those acting on sulfuric ester bonds. The systematic name of this enzyme class is ''N''-acetyl-D-galactosamine-6-sulfate 6-sulfohydrolase. Other names in common use include chondroitin sulfatase, chondroitinase, galactose-6-sulfate sulfatase, acetylgalactosamine 6-sulfatase, ''N''-acetylgalactosamine-6-sulfate sulfatase, and ''N''-acetylgalactosamine 6-sulfatase. This enzyme participates in glycosaminoglycan degradation and degradation of glycan structures. Deficiency Morquio syndrome is a rare birth defect caused by a deficiency in this essential enzyme. Treatment options include enzyme replacement therapy with ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase ( Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU). Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials. History BioMarin was founded in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr. with an investment of a $1.5 million from Glyko Biomedical and went public in 1999. Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting. Business development In 2002, BioMarin acquir ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C), but the agency also enforces other laws, notably Section 361 of the Public Health Service Act, as well as associated regulations. Much of this regulatory-enforcement work is not d ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Enzyme Replacement Therapy
Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease. ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking. ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency (ADA-SCID). Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation. History ERT was developed in 1964 by Christian de Duve and Roscoe Brady. Leading work was done on this subject at the Department of Physiology at the Univers ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Preliminary Injunction
An injunction is a legal and equitable remedy in the form of a special court order that compels a party to do or refrain from specific acts. ("The court of appeals ... has exclusive jurisdiction to enjoin, set aside, suspend (in whole or in part), or to determine the validity of...."); ("Limit on injunctive relief'); ''Jennings v. Rodriguez'', 583 U.S. ___, ___138 S.Ct. 830 851 (2018); '' Wheaton College v. Burwell''134 S.Ct. 2806 2810-11 (2014) ("Under our precedents, an injunction is appropriate only if (1) it is necessary or appropriate in aid of our jurisdiction, and (2) the legal rights at issue are indisputably clear.") (internal quotation marks and brackets omitted); '' Lux v. Rodrigues''561 U.S. 1306 1308 (2010); ''Correctional Services Corp. v. Malesko''534 U.S. 61 74 (2001) (stating that "injunctive relief has long been recognized as the proper means for preventing entities from acting unconstitutionally."); '' Nken v. Holder''556 U.S. 418(2009); see also ''Alli v. Dec ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Reimbursement
Reimbursement is the act of compensating someone for an out-of-pocket expense by giving them an amount of money equal to what was spent. Companies, governments and nonprofit organizations may compensate their employees or officers for necessary and reasonable expenses; under US law, these expenses may be deducted from taxes by the organization and treated as untaxed income for the recipient provided that accountability conditions are met. UK law provides for deductions for travel and subsistence. Reimbursement is also provided for supply, day care, mobile, medical, or education expenses, as determined by the payer. Similarly, a university, academic conference, or business conference may reimburse the expenses of an invited speaker or attendee. Reimbursement is also used in insurance, when a provider pays for expenses after they have been paid directly by the policy holder or another party. This is especially relevant in health insurance, due to urgency, high costs, and administ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Legal Proceeding
Legal proceeding is an activity that seeks to invoke the power of a tribunal in order to enforce a law. Although the term may be defined more broadly or more narrowly as circumstances require, it has been noted that " e term ''legal proceedings'' includes proceedings brought by or at the instigation of a public authority, and an appeal against the decision of a court or tribunal". Legal proceedings are generally characterized by an orderly process in which participants or their representatives are able to present evidence in support of their claims, and to argue in favor of particular interpretations of the law, after which a judge, jury, or other trier of fact makes a determination of the factual and legal issues. *Activities needed to have a court deem legal process to have been provided, such as through service of process. *Conduct of a trial, whether a lawsuit or civil trial, or a criminal trial. *Issuance and enforcement of court orders, including those imposing foreclosure o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
De Tijd
''De Tijd'' (, ''The Times'') is a Belgian newspaper that mainly focuses on business and economics. It is printed on salmon pink paper since May 2009, following the example of its colleagues ''Financial Times'', ''Het Financieele Dagblad'', '' FT Deutschland'' and many more. History and profile Established under the name of ''De Financieel-Economische Tijd'' (''"The Financial Economical Times"'') in 1968, in 2003 the paper was renamed as ''De Tijd''. ''De Tijd'' is owned by De Persgroep (50%) and Rossel (50%). The current editor-in-chief of ''De Tijd'' is Stephanie De Smedt. It is published in Dutch language. ''De Tijd'' is a typical financial daily, covering economy and business, financial markets and national and international politics. The paper is the main information source for Belgian managers (CIM-survey 2009). It is published from Tuesday to Saturday with no Sunday or Monday edition. The weekend edition is enriched with two magazines: Netto, on personal finance, and Saba ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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