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Omaveloxolone
Omaveloxolone, sold under the brand name Skyclarys, is a medication used for the treatment of Friedreich's ataxia. It is taken by mouth. The most common side effects include an increase in alanine transaminase and an increase of aspartate aminotransferase, which can be signs of liver damage, headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain. Omaveloxolone was approved for medical use in the United States in February 2023, and in the European Union in February 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Omaveloxolone is indicated for the treatment of Friedreich's ataxia. Friedreich's ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, poor balance, difficulty walkin ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Friedreich's Ataxia
Friedreich's ataxia (FRDA) is a rare, inherited, autosomal recessive neurodegenerative disorder that primarily affects the nervous system, causing progressive damage to the spinal cord, peripheral nerves, and cerebellum, leading to impaired muscle coordination (''ataxia''). The condition typically manifests in childhood or adolescence, with initial symptoms including difficulty walking, loss of balance, and poor coordination. As the disease progresses, it can also impact speech, vision, and hearing. Many individuals with Friedreich's ataxia develop scoliosis, diabetes, and hypertrophic cardiomyopathy, a serious heart condition that is a leading cause of mortality in patients. Friedreich's ataxia is caused by mutations in the ''FXN'' gene, which result in reduced production of frataxin, a protein essential for mitochondrial function, particularly in iron-sulfur cluster biogenesis. The deficiency of frataxin disrupts cellular energy production and leads to oxidative stress, co ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Reata Pharmaceuticals
Reata Pharmaceuticals, Inc. is a pharmaceutical company based in Plano, Texas. Founded in 2002, it is primarily focused on investigating experimental oral antioxidative and anti-inflammatory drugs, which dually activate the antioxidative transcription factor Nrf2 and inhibit the pro-inflammatory transcription factor NF-κB. The last CEO of Reata Pharmaceuticals was J. Warren Huff. In July 2023, it was announced Reata had been acquired by the Cambridge, Massachusetts-headquartered multinational biotechnology company, Biogen for nearly $6.5 billion. The purchase was completed on September 26 and Reata stock was delisted from the Nasdaq. Pipeline The antioxidative and anti-inflammatory compounds bardoxolone methyl and RTA 408 are the lead clinical development compounds in Reata’s portfolio. Bardoxolone Methyl Bardoxolone methyl was one of the first of the class of synthetic triterpenoids to be studied in the clinic. It has been evaluated in Phase 1 studies for cancer, Phase ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
By Mouth
Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administration can be easier and less painful than other routes of administration, such as Injection (medicine), injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mouth". The expression is used in medicine to describe a treatment that is taken orally (but not ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bioenergetics
Bioenergetics is a field in biochemistry and cell biology that concerns energy flow through living systems. This is an active area of biological research that includes the study of the transformation of energy in living organisms and the study of thousands of different cellular processes such as cellular respiration and the many other metabolic and enzymatic processes that lead to production and utilization of energy in forms such as adenosine triphosphate (ATP) molecules. Nelson, David L., Cox, Michael M. ''Lehninger: Principles of Biochemistry.'' New York: W.H. Freeman and Company, 2013. Sixth ed., pg. 27. That is, the goal of bioenergetics is to describe how living organisms acquire and transform energy in order to perform biological work. Nelson, David L., Cox, Michael M. ''Lehninger: Principles of Biochemistry.'' New York: W.H. Freeman and Company, 2013. Sixth ed., pg. 24. The study of metabolic pathways is thus essential to bioenergetics. Overview Bioenergetics is the ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Committee For Medicinal Products For Human Use
The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding medicinal product Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy ( pharmacotherapy) is an important part of the ...s for human use. See also * Committee for Medicinal Products for Veterinary Use References External links Committee for Medicinal Products for Human Use (CHMP) Health and the European Union {{eu-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Priority Review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability. Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a fee (approximately $2.8 million) to use the voucher. When seeking approval for a drug, manufacturers can apply to the FDA for priority review. This is granted when a drug is intended to treat a serious condition and would "provide a sig ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fast Track (FDA)
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and attempt to make a decision within sixty days. Purpose Fast track is one of five FDA approaches to make new drugs available as rapidly as possible: the others are priority review, breakthrough therapy, accelerated approval and regenerative medicine advanced therapy. Fast track was introduced by the FDA Modernization Act of 1997. Requirements Fast track designation is designed to aid in the development and expedite the review of drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Serious condition: det ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug
An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug medical research, research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to en ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cataract Surgery
Cataract surgery, also called lens replacement surgery, is the removal of the natural lens (anatomy), lens of the human eye, eye that has developed a cataract, an opaque or cloudy area. The eye's natural lens is usually replaced with an artificial intraocular lens (IOL) implant. Over time, metabolic changes of the crystalline lens fibres lead to the development of a cataract, causing impairment or loss of vision. Some infants are born with congenital cataracts, and environmental factors may lead to cataract formation. Early symptoms may include strong Glare (vision), glare from lights and small light sources at night and reduced visual acuity at low light levels. During cataract surgery, the cloudy natural lens is removed from the posterior chamber, either by emulsification in place or by cutting it out. An IOL is usually implanted in its place (PCIOL), or less frequently in front of the chamber, to restore useful focus. Cataract surgery is generally performed by an ophthalmolo ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Corneal Endothelium
The corneal endothelium is a single layer of endothelial cells on the inner surface of the cornea. It faces the chamber formed between the cornea and the iris. The corneal endothelium are specialized, flattened, mitochondria-rich cells that line the posterior surface of the cornea and face the anterior chamber of the eye. The corneal endothelium governs fluid and solute transport across the posterior surface of the cornea and maintains the cornea in the slightly dehydrated state that is required for optical transparency. Embryology and anatomy The corneal endothelium is embryologically derived from the neural crest. The postnatal total endothelial cellularity of the cornea (approximately 300,000 cells per cornea) is achieved as early as the second trimester of gestation. Thereafter the endothelial cell density (but not the absolute number of cells) rapidly declines, as the fetal cornea grows in surface area, achieving a final adult density of approximately 2400 - 3200 cel ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunooncology
Cancer immunotherapy (immuno-oncotherapy) is the stimulation of the immune system to treat cancer, improving the immune system's natural ability to fight the disease. It is an application of the fundamental research of cancer immunology (immuno-oncology) and a growing subspecialty of oncology. Cancer immunotherapy exploits the fact that cancer cells often have tumor antigens, molecules on their surface that can bind to antibody proteins or T-cell receptors, triggering an immune system response. The tumor antigens are often proteins or other macromolecules (e.g., carbohydrates). Normal antibodies bind to external pathogens, but the modified immunotherapy antibodies bind to the tumor antigens marking and identifying the cancer cells for the immune system to inhibit or kill. The clinical success of cancer immunotherapy is highly variable between different forms of cancer; for instance, certain subtypes of gastric cancer react well to the approach whereas immunotherapy is not effect ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Mitochondrial Myopathies
Mitochondrial myopathies are types of myopathies associated with mitochondrial disease. Adenosine triphosphate ( ATP), the chemical used to provide energy for the cell, cannot be produced sufficiently by oxidative phosphorylation when the mitochondrion is either damaged or missing necessary enzymes or transport proteins. With ATP production deficient in mitochondria, there is an over-reliance on anaerobic glycolysis which leads to lactic acidosis either at rest or exercise-induced. Primary mitochondrial myopathies are inherited, while secondary mitochondrial myopathies may be inherited (e.g. Duchenne's muscular dystrophy) or environmental (e.g. alcoholic myopathy). When it is an inherited primary disease, it is one of the metabolic myopathies. On biopsy, the muscle tissue of patients with these diseases usually demonstrate "ragged red" muscle fibers on Gomori trichrome staining. The ragged-red appearance is due to a buildup of abnormal mitochondria underneath the plasma membrane. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
