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ATC Code A16
A16A Other alimentary tract and metabolism products A16AA Amino acids and derivatives :A16AA01 Levocarnitine :A16AA02 Ademetionine :A16AA03 Levoglutamide :A16AA04 Mercaptamine :A16AA05 Carglumic acid :A16AA06 Betaine :A16AA07 Metreleptin :QA16AA51 Levocarnitine, combinations A16AB Enzymes :A16AB01 Alglucerase :A16AB02 Imiglucerase :A16AB03 Agalsidase alfa :A16AB04 Agalsidase beta :A16AB05 Laronidase :A16AB06 Sacrosidase :A16AB07 Alglucosidase alfa :A16AB08 Galsulfase :A16AB09 Idursulfase :A16AB10 Velaglucerase alfa :A16AB11 Taliglucerase alfa :A16AB12 Elosulfase alfa :A16AB13 Asfotase alfa :A16AB14 Sebelipase alfa :A16AB15 Velmanase alfa :A16AB16 Idursulfase beta :A16AB17 Cerliponase alfa :A16AB18 Vestronidase alfa :A16AB19 Pegvaliase :A16AB20 Pegunigalsidase alfa :A16AB21 Atidarsagene autotemcel :A16AB22 Avalglucosidase alfa :A16AB23 Cipaglucosidase alfa :A16AB24 Pegzilarginase :A16AB25 Olipudase alfa A16AX Various alimentary tract and metabolism products :A16AX01 Thi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Amino Acid
Amino acids are organic compounds that contain both amino and carboxylic acid functional groups. Although hundreds of amino acids exist in nature, by far the most important are the alpha-amino acids, which comprise proteins. Only 22 alpha amino acids appear in the genetic code. Amino acids can be classified according to the locations of the core structural functional groups, as Alpha and beta carbon, alpha- , beta- , gamma- or delta- amino acids; other categories relate to Chemical polarity, polarity, ionization, and side chain group type (aliphatic, Open-chain compound, acyclic, aromatic, containing hydroxyl or sulfur, etc.). In the form of proteins, amino acid '' residues'' form the second-largest component (water being the largest) of human muscles and other tissues. Beyond their role as residues in proteins, amino acids participate in a number of processes such as neurotransmitter transport and biosynthesis. It is thought that they played a key role in enabling life ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Idursulfase
Idursulfase (brand name Elaprase), manufactured by Takeda, is a drug used to treat Hunter syndrome (also called MPS-II). It is a purified form of the lysosomal enzyme iduronate-2-sulfatase and is produced by recombinant DNA technology Molecular cloning is a set of experimental methods in molecular biology that are used to assemble recombinant DNA molecules and to direct their replication within host organisms. The use of the word ''cloning'' refers to the fact that the metho ... in a human cell line. It is one of the most expensive drugs ever produced, costing US$567,412 per patient per year. Barbara Kollmeyer, Marketwatch, Fed. 3, 2016 References External links *[...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Avalglucosidase Alfa
Avalglucosidase alfa, sold under the brand name Nexviazyme, is an enzyme replacement therapy medication used for the treatment of glycogen storage disease type II (Pompe disease). The most common side effects include headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), skin redness (erythema), feeling of "pins and needles" (paresthesia) and skin welts (urticaria). People with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which causes muscle weakness and premature death from respiratory or heart failure. Avalglucosidase alfa was approved for medical use in the United States in August 2021, and in the European Union in June 2022. Medical uses Avalglucosidase alfa is indicated for the treatment of people aged one year and older with late-onset Pompe disease (lysosomal acid alpha-g ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Atidarsagene Autotemcel
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy treatment for metachromatic leukodystrophy (MLD) developed by Orchard Therapeutics. It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A Arylsulfatase A (or cerebroside-sulfatase) is an enzyme that breaks down sulfatides, namely cerebroside 3-sulfate into cerebroside and sulfate. In humans, arylsulfatase A is encoded by the ''ARSA'' gene. Pathology A deficiency is associated with m ... (''ARSA'') gene. Atidarsagene autotemcel was approved for medical use in the European Union in December 2020, Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. and in the United Kingdom in February 2021. Medical uses Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pegunigalsidase Alfa
Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease. It is a recombinant human α-galactosidase-A. It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. The most common side effects are infusion-related reactions, hypersensitivity and asthenia. Pegunigalsidase alfa was approved for medical use in both the European Union and the United States in May 2023. Medical uses Pegunigalsidase alfa is indicated for long-term enzyme replacement therapy in aduls with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Society and culture Legal status On 23 February 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency The European Medicines Agency (EMA) is an agency of the Europe ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pegvaliase
Pegvaliase, sold under the brand name Palynziq, is a medication for the treatment of the genetic disease phenylketonuria. Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels. It was approved by the Food and Drug Administration for use in the United States in 2018. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication A first-in-class medication is a pharmaceutical that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medicat .... References External links * Drugs acting on the gastrointestinal system and metabolism Recombinant proteins Orphan drugs {{gastrointestinal-drug-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Vestronidase Alfa
Vestronidase alfa, sold under brand name Mepsevii, is a drug for the treatment of Sly syndrome. It is a recombinant form of the human enzyme beta-glucuronidase. It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs. The most common side effects after treatment with vestronidase alfa include infusion site reactions, diarrhea, rash (urticaria) and anaphylaxis (sudden, severe allergic reaction). The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. It was approved for use in the European Union in August 2018. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Medical uses Mepsevii is indicated for the treatment of non-neurol ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cerliponase Alfa
Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, a neurodegenerative lysosomal storage disease. Specifically, Cerliponase alfa is meant to slow loss of motor function in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency, a soluble lysosomal enzyme deficiency. Approved by the United States Food and Drug Administration (FDA) on 27 April 2017, this is the first treatment for a neuronal ceroid lipofuscinosis of its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. History TPP1 was identified as the enzyme deficient in CLN2 Batten disease in 1997, via biochemical analysis that identified proteins missing a mannose-6-phosphate lysosoma ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Idursulfase Beta
Idursulfase (brand name Elaprase), manufactured by Takeda, is a drug used to treat Hunter syndrome (also called MPS-II). It is a purified form of the lysosomal enzyme iduronate-2-sulfatase and is produced by recombinant DNA technology Molecular cloning is a set of experimental methods in molecular biology that are used to assemble recombinant DNA molecules and to direct their replication within host organisms. The use of the word ''cloning'' refers to the fact that the metho ... in a human cell line. It is one of the most expensive drugs ever produced, costing US$567,412 per patient per year. Barbara Kollmeyer, Marketwatch, Fed. 3, 2016 References External links *[...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Velmanase Alfa
Velmanase alfa, sold under the brand name Lamzede, is a medication used for the treatment of alpha-mannosidosis. Velmanase alfa is a recombinant human lysosomal alpha-mannosidase. The most common adverse reactions include hypersensitivity reactions including anaphylaxis, a severe, potentially life-threatening allergic reaction. Velmanase alfa was approved for medical use in the European Union in March 2018, and in the United States in February 2023. Velmanase alfa is the first enzyme replacement therapy approved in the US for the treatment of the non-central nervous system manifestations of alpha-mannosidosis. Medical uses Velmanase alfa is indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ... for the treatment of the non-central nervous system manifestations of alpha- ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sebelipase Alfa
Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the enzyme lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D). Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. It is administered via intraveneous infusion. It was approved for medical use in the European Union and in the United States in 2015. Medical uses Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency. History Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production, chickens are genetically modified to produce the recombinant form of LAL (rhLAL) in their egg white. After extraction and purification it becomes available as the medication. On 8 December 2015 the FDA announced that its approval came ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Asfotase Alfa
Asfotase alfa, sold under the brand name Strensiq, is a medication used in the treatment of people with perinatal/infantile- and juvenile-onset hypophosphatasia. U.S. Patent 7,763,712 The most common side effects include injection site reactions, hypersensitivity reactions (such as difficulty breathing, nausea, dizziness and fever), lipodystrophy (a loss of fat tissue resulting in an indentation in the skin or a thickening of fat tissue resulting in a lump under the skin) at the injection site, and ectopic calcifications of the eyes and kidney. The enzyme tissue non-specific alkaline phosphat ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
