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Ultragenyx
Ultragenyx is an American biopharmaceutical company involved in the Research and Development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases. Ultragenyx is based in Novato, CA and Brisbane, CA and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. The company’s Latin American headquarters is located in Miami. Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo. Ultragenyx has three products Burosumab, Triheptanoin and Vestronidase alfa that have received FDA approval and several others currently in clinical trials. The company also holds th ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Triheptanoin
Triheptanoin, sold under the brand name Dojolvi, is a medication for the treatment of children and adults with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). The most common adverse reactions include abdominal pain, diarrhea, vomiting, and nausea. Triheptanoin was approved for medical use in the United States in June 2020. Triheptanoin is a triglyceride that is composed of three seven-carbon (C7:0) fatty acids. These odd-carbon fatty acids are able to provide anaplerotic substrates for the TCA cycle. Triheptanoin is used clinically in humans to treat inherited metabolic diseases, such as pyruvate carboxylase deficiency and carnitine palmitoyltransferase II deficiency. It also appears to increase the efficacy of the ketogenic diet as a treatment for epilepsy. Since triheptanoin is composed of odd-carbon fatty acids, it can produce ketone bodies with five carbon atoms, as opposed to even-carbon fatty acids which are metabolized to ketone bodies with ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Osteogenesis Imperfecta
Osteogenesis imperfecta (; OI), colloquially known as brittle bone disease, is a group of genetic disorders that all result in bones that break easily. The range of symptoms—on the skeleton as well as on the body's other organs—may be mild to severe. Symptoms found in various types of OI include whites of the eye (sclerae) that are blue instead, short stature, loose joints, hearing loss, breathing problems and problems with the teeth (dentinogenesis imperfecta). Potentially life-threatening complications, all of which become more common in more severe OI, include: tearing ( dissection) of the major arteries, such as the aorta; pulmonary valve insufficiency secondary to distortion of the ribcage; and basilar invagination. The underlying mechanism is usually a problem with connective tissue due to a lack of, or poorly formed, type I collagen. In more than 90% of cases, OI occurs due to mutations in the ''COL1A1'' or ''COL1A2'' genes. These mutations may be inherited ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Emil Kakkis
Emil Kakkis (born 1960) is an American medical geneticist known for his work to develop treatments for ultra rare disorders. He is the Founder of the Everylife Foundation for Rare Disease and Founder, CEO and President of Ultragenyx Pharmaceutical Inc. Professional background Kakkis began his work at Harbor–UCLA Medical Center working with minimal funding and support to develop an enzyme replacement therapy (Aldurazyme) for the rare disorder Mucopolysaccharidosis (MPS I). The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of a patient organization formed by Mark and Jeanne Dant for their son Ryan, called the Ryan Foundation. Aldurazyme development was later supported by BioMarin and eventually, their partner Genzyme leading to U.S. Food and Drug Administration (FDA) approval in 2003. During his tenure at BioMarin, Kakkis guided the development and approval of two more treatments for rare di ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Novato, California
Novato (Spanish for "Novatus") is a city in Marin County, California, in the North Bay region of the Bay Area. At the 2020 census, Novato had a population of 53,225. History What is now Novato was originally the site of several Coast Miwok villages: Chokecherry, near downtown Novato; Puyuku, near Ignacio; and Olómpali, at the present-day Olompali State Historic Park. Mexican era In 1839, the Mexican government granted the Rancho Novato to Fernando Feliz. The rancho was named after a local Miwok leader who had probably been given the name of Saint Novatus at his baptism. Subsequently, four additional land grants were made in the area: Rancho Corte Madera de Novato, to John Martin in 1839; Rancho San Jose, to Ignacio Pacheco in 1840; Rancho Olómpali, awarded in 1843 to Camilo Ynitia, son of a Coast Miwok chief; and Rancho Nicasio, by far the largest at , awarded to Pablo de la Guerra and John B.R. Cooper in 1844. Post-Conquest era Following the American Conquest of C ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Arcturus Therapeutics
Arcturus Therapeutics is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed a novel, potent, and safe RNA therapeutics platform called Lunar, a proprietary lipid-enabled delivery system for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides (ASO), and microRNA. The company's pipeline includes RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency,and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the preclinical development phase. Company history Founded in 2013 by Joseph Payne and Dr. Pad Chivukula, Arcturus Therapeutics is headquartered in San Diego, California, USA. The company's core technology platform is a proprietary lipid n ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Burosumab
Burosumab, sold under the brand name Crysvita, is a human monoclonal antibody medication approved 2018 for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia. Medical uses In the European Union and the United States, burosumab is indicated for the treatment of adults and children ages one year and older with X-linked hypophosphatemia (XLH), a rare, inherited form of rickets. caused by overproduction of a hormone called FGF23 (fibroblast growth factor 23) in bone cells. FGF23 is responsible for blocking phosphate re-absorption in the kidney and the suppression of the vitamin D dependent phosphate absorption in the intestine. Due to the excess activity of FGF23, phosphate levels in the blood are abnormally low (hypophosphatemia), which affects the constitution of bone. Thus, burosumab is designed to bind to the FGF23 receptor and inhibit the excess activity of the FGF23 hormone within the body. In the United States, burosumab is also approved to treat pe ... [...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] |
Mucopolysaccharidosis
Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosome, lysosomal enzymes needed to break down molecules called glycosaminoglycans (GAGs). These long chains of sugar carbohydrates occur within the Cell (biology), cells that help build bone, cartilage, tendons, corneas, skin and connective tissue. GAGs (formerly called mucopolysaccharides) are also found in Synovial fluid, the fluids that lubricate joints. Individuals with mucopolysaccharidosis either do not produce enough of one of the eleven enzymes required to break down these sugar chains into simpler molecules, or they produce enzymes that do not work properly. Over time, these GAGs collect in the cells, blood and connective tissues. The result is permanent, progressive cellular damage which affects appearance, physical abilities, organ and system functioning. The mucopolysaccharidoses are part of the lysosomal storage disease family, a group of more than 40 genetic disord ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Russell 1000
The Russell 1000 Index is a stock market index that tracks the highest-ranking 1,000 stocks in the Russell 3000 Index, which represent about 93% of the total market capitalization of that index. , the stocks of the Russell 1000 Index had a weighted average market capitalization of $608.1 billion and a median market capitalization of $15.1 billion. , components ranged in market capitalization from $1.8 billion to $1.4 trillion. The index, which was launched on January 1, 1984, is maintained by FTSE Russell, a subsidiary of the London Stock Exchange Group. The ticker symbol is ^RUI. There are several exchange-traded funds and mutual funds that track the index. Record values Annual returns Top sectors by weight *Technology *Consumer Discretionary * Health Care *Industrials *Financial services Top 10 holdings *Apple () *Microsoft () *Amazon () *Alphabet (Class A) () * Tesla () *Alphabet (Class C) () * Meta () *Nvidia () *Berkshire Hathaway () *UnitedHealth Group () (as of Dece ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adeno-associated Virus
Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus ''Dependoparvovirus'', which in turn belongs to the family ''Parvoviridae''. They are small (approximately 26 nm in diameter) replication-defective, nonenveloped viruses and have linear single-stranded DNA (ssDNA) genome of approximately 4.8 kilobases (kb). AAV are not currently known to cause disease. The viruses cause a very mild immune response. Several additional features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell. In the native virus, however, integration of virally carried genes into the host genome does occur. Integration can be important for certain applications, but can also have unwan ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hereditary Inclusion Body Myopathy
Hereditary inclusion body myopathies (HIBM) are a group of rare genetic disorders which have different symptoms. Generally, they are neuromuscular disorders characterized by muscle weakness developing in young adults. Hereditary inclusion body myopathies comprise both autosomal recessive and autosomal dominant muscle disorders that have a variable expression (phenotype) in individuals, but all share similar structural features in the muscles. HIBMs are a group of muscle wasting disorders that are uncommon in the general world population. One autosomal recessive form of HIBM is known as IBM2 or GNE myopathy, which is a common genetic disorder amongst people of Iranian Jewish descent. IBM2 has also been identified in other minorities throughout the world, including those of Asian, European, and South American, and Middle Eastern descent. In Japan and other East Asian countries, this disorder is known as Distal Myopathy with Rimmed Vacuoles (DMRV). IBM2 causes progressive muscle ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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