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Swedish Orphan Biovitrum
Swedish Orphan Biovitrum AB is an international biopharmaceutical company dedicated to treatments in the areas of haematology, immunology and specialty care, based in Stockholm, Sweden. In 2020 it had a revenue of SEK 15.261 billion and 1,509 employees. History Sobi traces its origins to a subsidiary of Kärnbolaget Aktiebolag Biokemisk Industri in the 1930s, which changed its name to in 1951 and merged with to become KabiVitrum in the 1970s. Sobi has been involved in the process development and manufacturing of recombinant protein drugs since the technology was first developed around 30 years ago, then as part of Kabivitrum, KabiVitrum. Biovitrum was formed in 2001 through the merger of several units of Pharmacia (now Pfizer) and spun off to a consortium of investors led by Nordic Capital and MPM Capital Funds. Operations included a research unit focused on metabolic diseases, a process development unit for protein drugs and a plasma product operation. In 2002 Sobi sol ...
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Public Company
A public company is a company whose ownership is organized via shares of stock which are intended to be freely traded on a stock exchange or in over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (listed company), which facilitates the trade of shares, or not (unlisted public company). In some jurisdictions, public companies over a certain size must be listed on an exchange. In most cases, public companies are ''private'' enterprises in the ''private'' sector, and "public" emphasizes their reporting and trading on the public markets. Public companies are formed within the legal systems of particular states, and therefore have associations and formal designations which are distinct and separate in the polity in which they reside. In the United States, for example, a public company is usually a type of corporation (though a corporation need not be a public company), in the United Kingdom it is usually a public limited company (plc), i ...
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Biovitrum
Swedish Orphan Biovitrum AB is an international biopharmaceutical company dedicated to treatments in the areas of haematology, immunology and specialty care, based in Stockholm, Sweden. In 2020 it had a revenue of SEK 15.261 billion and 1,509 employees. History Sobi traces its origins to a subsidiary of Kärnbolaget Aktiebolag Biokemisk Industri in the 1930s, which changed its name to in 1951 and merged with to become KabiVitrum in the 1970s. Sobi has been involved in the process development and manufacturing of recombinant protein drugs since the technology was first developed around 30 years ago, then as part of KabiVitrum. Biovitrum was formed in 2001 through the merger of several units of Pharmacia (now Pfizer) and spun off to a consortium of investors led by Nordic Capital and MPM Capital Funds. Operations included a research unit focused on metabolic diseases, a process development unit for protein drugs and a plasma product operation. In 2002 Sobi sold its plas ...
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Hemophagocytic Lymphohistiocytosis
Hemophagocytic lymphohistiocytosis (HLH), also known as haemophagocytic lymphohistiocytosis (British spelling), and hemophagocytic or haemophagocytic syndrome, is an uncommon hematologic disorder seen more often in children than in adults. It is a life-threatening disease of severe hyperinflammation caused by uncontrolled proliferation of activated lymphocytes and macrophages, characterised by proliferation of morphologically benign lymphocytes and macrophages that secrete high amounts of inflammatory cytokines. It is classified as one of the cytokine storm syndromes. There are inherited and non-inherited (acquired) causes of hemophagocytic lymphohistiocytosis (HLH). Signs and symptoms The onset of HLH occurs before the age of one year in approximately 70 percent of cases. Familial HLH should be suspected if siblings are diagnosed with HLH or if symptoms recur when therapy has been stopped. Familial HLH is an autosomal recessive disease, hence each sibling of a child with familial ...
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Gamifant
Emapalumab, sold under the brand name Gamifant, is an anti- interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH), which has no cure. The most common side effects include infections, hypertension, infusion-related reactions, and pyrexia. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Emapalumab is used to treat primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Adverse effects In the clinical trials that lead to emapalumab's FDA approval, the most commonly reported adverse effects were infections (56%), high blood pressure (41%), infusion reactions (27%), and fever (24%). Serious adverse effects occurred in about half of the subjects studied in the clinical trial that led to its FDA approval. Pharmacology Mechanism of action In the setting of HLH, over-secretion of I ...
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Respiratory Syncytial Virus
Respiratory syncytial virus (RSV), also called human respiratory syncytial virus (hRSV) and human orthopneumovirus, is a common, contagious virus that causes infections of the respiratory tract. It is a negative-sense, single-stranded RNA virus. Its name is derived from the large cells known as ''syncytia'' that form when infected cells fuse. RSV is the single most common cause of respiratory hospitalization in infants, and reinfection remains common in later life: it is a notable pathogen in all age groups. Infection rates are typically higher during the cold winter months, causing bronchiolitis in infants, common colds in adults, and more serious respiratory illnesses such as pneumonia in the elderly and immunocompromised. RSV can cause outbreaks both in the community and in hospital settings. Following initial infection via the eyes or nose, the virus infects the epithelial cells of the upper and lower airway, causing inflammation, cell damage, and airway obstruction. A variet ...
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Synagis
Palivizumab, sold under the brand name Synagis, is a monoclonal antibody produced by recombinant DNA technology used to prevent severe disease caused by respiratory syncytial virus (RSV) infections. It is recommended for infants at high-risk for RSV due to conditions such as prematurity or other medical problems including heart or lung diseases. The most common side effects include fever and rash. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Palivizumab is a humanized monoclonal antibody (IgG) directed against an epitope in the A antigenic site of the F protein of RSV. In two phase III clinical trials in the pediatric population, palivizumab reduced the risk of hospitalization due to RSV infection by 55% and 45%. Palivizumab is dosed once a month via intramuscular (IM) injection, to be administered throughout the duration of the RSV season, which in based on past trends has started in Mi ...
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Kineret (medication)
Anakinra, sold under the brand name Kineret, is a biopharmaceutical medication used to treat rheumatoid arthritis, cryopyrin-associated periodic syndromes, familial Mediterranean fever, and Still's disease. It is a recombinant and slightly modified version of the human interleukin 1 receptor antagonist protein. It is marketed by Swedish Orphan Biovitrum. Anakinra is administered by subcutaneous injection. Medical uses It is used as a second line treatment to manage symptoms of rheumatoid arthritis after treatment with a disease-modifying antirheumatic drug (DMARD) has failed. It can be used in combination with some DMARDs. It is used to people with a cryopyrin-associated periodic syndrome, including neonatal-onset multisystem inflammatory disease. It is used to treat Schnitzler's syndrome (off label in the US). Its response rate is such that it has been suggested that "Treatment failures should lead to reconsider the diagnosis." Off label, it is used to treat systemic juve ...
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Chronic Liver Disease
Chronic liver disease in the clinical context is a disease process of the liver that involves a process of progressive destruction and regeneration of the liver parenchyma leading to fibrosis and cirrhosis. "Chronic liver disease" refers to disease of the liver which lasts over a period of six months. It consists of a wide range of liver pathologies which include inflammation (chronic hepatitis), liver cirrhosis, and hepatocellular carcinoma. The entire spectrum need not be experienced. Signs and symptoms Signs of chronic liver disease detectable on clinical examination can be divided into those that are associated with the diagnosis of chronic liver disease, associated with decompensation, and associated with the cause. Chronic liver disease * Nail clubbing * Palmar erythema * Spider nevi (angiomata) * Gynaecomastia * Feminising hair distribution * Testicular atrophy * Small irregular shrunken liver * Anaemia * Caput medusae Decompensation * Drowsiness (encephalopathy) * Hyperve ...
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Immune Thrombocytopenic Purpura
Immune thrombocytopenic purpura (ITP), also known as idiopathic thrombocytopenic purpura or immune thrombocytopenia, is a type of thrombocytopenic purpura defined as an isolated low platelet count with a normal bone marrow in the absence of other causes of low platelets. It causes a characteristic red or purple bruise-like rash and an increased tendency to bleed. Two distinct clinical syndromes manifest as an acute condition in children and a chronic condition in adults. The acute form often follows an infection and spontaneously resolves within two months. Chronic immune thrombocytopenia persists longer than six months with a specific cause being unknown. ITP is an autoimmune disease with antibodies detectable against several platelet surface structures. ITP is diagnosed by identifying a low platelet count on a complete blood count (a common blood test). However, since the diagnosis depends on the exclusion of other causes of a low platelet count, additional investigations (su ...
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Doptelet
Avatrombopag, sold under the brand name Doptelet, is a medication that used for certain conditions that lead to thrombocytopenia (low platelets) such as thrombocytopenia associated with chronic liver disease in adults who are to undergo a planned medical or dental procedure. It was approved for medical use in the United States in May 2018, the European Union in June 2019, and Australia in January 2023. It acts as a thrombopoietin receptor The thrombopoietin receptor also known as the myeloproliferative leukemia protein or CD110 (Cluster of Differentiation 110) is a protein that in humans is encoded by the MPL (myeloproliferative leukemia virus) oncogene. Discovery In 1990 an on ... agonist. References External links * Drugs acting on the blood and blood forming organs Orphan drugs Thrombopoietin receptor agonists Thiophenes Chloropyridines Thiazoles Piperazines {{blood-drug-stub ...
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Haemophilia
Haemophilia, or hemophilia (), is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or a decreased level of consciousness. There are two main types of haemophilia: haemophilia A, which occurs due to low amounts of clotting factor VIII, and haemophilia B, which occurs due to low levels of clotting factor IX. They are typically inherited from one's parents through an X chromosome carrying a nonfunctional gene. Rarely a new mutation may occur during early development or haemophilia may develop later in life due to antibodies forming against ...
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Alprolix
Factor IX (or Christmas factor) () is one of the serine proteases of the coagulation system; it belongs to peptidase family S1. Deficiency of this protein causes haemophilia B. It was discovered in 1952 after a young boy named Stephen Christmas was found to be lacking this exact factor, leading to haemophilia. Coagulation factor IX is on the World Health Organization's List of Essential Medicines. Physiology Factor IX is produced as a zymogen, an inactive precursor. It is processed to remove the signal peptide, glycosylated and then cleaved by factor XIa (of the contact pathway) or factor VIIa (of the tissue factor pathway) to produce a two-chain form, where the chains are linked by a disulfide bridge. When activated into factor IXa, in the presence of Ca2+, membrane phospholipids, and a Factor VIII cofactor, it hydrolyses one arginine- isoleucine bond in factor X to form factor Xa. Factor IX is inhibited by antithrombin. Factor IX expression increases with age in hu ...
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