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| Formula | C23H28N2O |
| Molar mass | 348.490 g·mol−1 |
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Iferanserin (INN; VEN-309) is a drug which acts as a selective 5-HT2A receptor antagonist. [1] It is under development as an intra-rectal formulation for the treatment of hemorrhoid disease, and as of February 2012, is in phase IIb clinical trials. [1]
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time.
Constipation refers to bowel movements that are infrequent or hard to pass. The stool is often hard and dry. Other symptoms may include abdominal pain, bloating, and feeling as if one has not completely passed the bowel movement. Complications from constipation may include hemorrhoids, anal fissure or fecal impaction. The normal frequency of bowel movements in adults is between three per day and three per week. Babies often have three to four bowel movements per day while young children typically have two to three per day.
Hemorrhoids, also known as piles, are vascular structures in the anal canal. In their normal state, they are cushions that help with stool control. They become a disease when swollen or inflamed; the unqualified term "hemorrhoid" is often used to refer to the disease. The signs and symptoms of hemorrhoids depend on the type present. Internal hemorrhoids often result in painless, bright red rectal bleeding when defecating. External hemorrhoids often result in pain and swelling in the area of the anus. If bleeding occurs, it is usually darker. Symptoms frequently get better after a few days. A skin tag may remain after the healing of an external hemorrhoid.
Clinical trials are experiments or observations done in clinical research. Such prospective biomedical or behavioral research studies on human participants are designed to answer specific questions about biomedical or behavioral interventions, including new treatments and known interventions that warrant further study and comparison. Clinical trials generate data on dosage, safety and efficacy. They are conducted only after they have received health authority/ethics committee approval in the country where approval of the therapy is sought. These authorities are responsible for vetting the risk/benefit ratio of the trial—their approval does not mean the therapy is 'safe' or effective, only that the trial may be conducted.
Osteoarthritis (OA) is a type of degenerative joint disease that results from breakdown of joint cartilage and underlying bone. The most common symptoms are joint pain and stiffness. Usually the symptoms progress slowly over years. Initially they may occur only after exercise but can become constant over time. Other symptoms may include joint swelling, decreased range of motion, and, when the back is affected, weakness or numbness of the arms and legs. The most commonly involved joints are the two near the ends of the fingers and the joint at the base of the thumbs; the knee and hip joints; and the joints of the neck and lower back. Joints on one side of the body are often more affected than those on the other. The symptoms can interfere with work and normal daily activities. Unlike some other types of arthritis, only the joints, not internal organs, are affected.
A rectal prolapse occurs when walls of the rectum have prolapsed to such a degree that they protrude out of the anus and are visible outside the body. However, most researchers agree that there are 3 to 5 different types of rectal prolapse, depending on whether the prolapsed section is visible externally, and whether the full or only partial thickness of the rectal wall is involved.
Spinocerebellar ataxia (SCA) is a progressive, degenerative, genetic disease with multiple types, each of which could be considered a neurological condition in its own right. An estimated 150,000 people in the United States have a diagnosis of spinocerebellar ataxia at any given time. SCA is hereditary, progressive, degenerative, and often fatal. There is no known effective treatment or cure. SCA can affect anyone of any age. The disease is caused by either a recessive or dominant gene. In many cases people are not aware that they carry a relevant gene until they have children who begin to show signs of having the disorder.
Blood in stool looks different depending on how early it enters the digestive tract—and thus how much digestive action it has been exposed to—and how much there is. The term can refer either to melena, with a black appearance, typically originating from upper gastrointestinal bleeding; or to hematochezia, with a red color, typically originating from lower gastrointestinal bleeding. Evaluation of the blood found in stool depends on its characteristics, in terms of color, quantity and other features, which can point to its source, however, more serious conditions can present with a mixed picture, or with the form of bleeding that is found in another section of the tract. The term "blood in stool" is usually only used to describe visible blood, and not fecal occult blood, which is found only after physical examination and chemical laboratory testing.
Lower gastrointestinal bleeding, commonly abbreviated LGIB, is any form of gastrointestinal bleeding in the lower gastrointestinal tract. LGIB is a common reason for seeking medical attention at a hospital's emergency department. LGIB accounts for 30–40% of all gastrointestinal bleeding and is less common than upper gastrointestinal bleeding (UGIB). It is estimated that UGIB accounts for 100–200 per 100,000 cases versus 20–27 per 100,000 cases for LGIB. Approximately 85% of lower gastrointestinal bleeding involves the colon, 10% are from bleeds that are actually upper gastrointestinal bleeds, and 3–5% involve the small intestines.
Diosmin, a flavone glycoside of diosmetin, is manufactured from citrus fruit peels as a phlebotonic non-prescription dietary supplement used to aid treatment of hemorrhoids or chronic venous diseases, mainly of the legs.
Aggressive fibromatosis or desmoid tumor is a rare condition. Desmoid tumors arise from cells called fibroblasts, which are found throughout the body and provide structural support, protection to the vital organs, and play a critical role in wound healing. These tumors tend to occur in women in their thirties, but can occur in anyone at any age. They can be either relatively slow-growing or malignant. However, aggressive fibromatosis is locally aggressive and can cause life-threatening problems or even death when they compress vital organs such as intestines, kidneys, lungs, blood vessels, or nerves. Most cases are sporadic, but some are associated with familial adenomatous polyposis (FAP). Approximately 10% of individuals with Gardner's syndrome, a type of FAP with extracolonic features, have desmoid tumors.
Stapled hemorrhoidopexy is a surgical procedure that involves the cutting and removal of anal hemorrhoidal vascular cushion, whose function is to help to seal stools and create continence. Procedure also removes abnormally enlarged hemorrhoidal tissue, followed by the repositioning of the remaining hemorrhoidal tissue back to its normal anatomic position. Severe cases of hemorrhoidal prolapse will normally require surgery. Newer surgical procedures include stapled transanal rectal resection (STARR) and procedure for prolapse and hemorrhoids (PPH). Both STARR and PPH are contraindicated in persons with either enterocele or anismus.
The GM2 gangliosidoses are a group of three related genetic disorders that result from a deficiency of the enzyme beta-hexosaminidase. This enzyme catalyzes the biodegradation of fatty acid derivatives known as gangliosides. The diseases are better known by their individual names: Tay–Sachs disease, AB variant, and Sandhoff disease.
Acetylleucine is a modified amino acid used in the treatment of vertigo and cerebellar ataxia.
In humans, the anus is the external opening of the rectum, located inside the intergluteal cleft and separated from the genitals by the perineum. Two sphincters control the exit of feces from the body during an act of defecation, which is the primary function of the anus. These are the internal anal sphincter and the external anal sphincter, which are circular muscles that normally maintain constriction of the orifice and which relaxes as required by normal physiological functioning. The inner sphincter is involuntary and the outer is voluntary. It is located behind the perineum which is located behind the vagina or scrotum.
Transanal hemorrhoidal dearterialization (THD) is a minimally invasive surgical procedure for the treatment of internal hemorrhoids.
Ruxolitinib, sold under the brand names Jakafi and Jakavi, is a medication for the treatment of intermediate or high-risk myelofibrosis, a type of myeloproliferative disorder that affects the bone marrow; polycythemia vera (PCV), when there has been an inadequate response to or intolerance of hydroxyurea; and steroid-refractory acute graft-versus-host disease. It was developed and marketed by Incyte Corp in the US under the brand name Jakafi, and by Novartis elsewhere in the world, under the brand name Jakavi. Ruxolitinib is a janus kinase inhibitor.
Sprifermin (INN), also known as recombinant human fibroblast growth factor 18 (rhFGF18), is a recombinant form of human fibroblast growth factor 18 (FGF18) which is under development by Merck and Nordic Bioscience for the treatment of osteoarthritis. As a recombinant form of FGF18, sprifermin is a potent agonist of the FGFR2 and FGFR3.
Verubecestat (MK-8931) was an experimental drug for the treatment of Alzheimer's disease. It is an inhibitor of beta-secretase 1 (BACE1), which, after initial promise proved disappointing.
Phenserine is a synthetic drug which has been investigated as a medication to treat Alzheimer's disease (AD), as the drug exhibits neuroprotective and neurotrophic effects.
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