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Nedosiran
Nedosiran, sold under the brand name Rivfloza, is a medication used for the treatment of primary hyperoxaluria. It is an LDHA-directed small interfering RNA developed by Dicerna Pharmaceuticals. The most common side effects include injection site reactions. Nedosiran was approved for medical use in the United States in September 2023. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Primary hyperoxaluria type 1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys. Nedosiran is indicated to lower urinary oxalate levels in people nine years of age and older with primary hyperoxaluria type 1 and relatively preserved kidney function. History The US FDA approved nedosiran based on evidence from a clinical trial which included 29 participants with primary hyperoxaluria type 1. Nedosiran was evaluated in one clinical trial of 29 participants with primary hyperoxaluria type 1 who were n ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Primary Hyperoxaluria
Primary hyperoxaluria is a rare condition (autosomal recessive) resulting in increased excretion of oxalate (up to 600 mg a day from normal 50 mg a day), with oxalate stones being common. Signs and symptoms Primary hyperoxaluria is an autosomal recessive disease, meaning both copies of the gene contain the mutation. Both parents must have one copy of this mutated gene to pass it on to their child, but they do not typically show signs or symptoms of the disease. A single kidney stone in children or recurrent stones in adults is often the first warning sign of primary hyperoxaluria. Other symptoms range from recurrent urinary tract infections, severe abdominal pain or pain in the side, blood in the urine, to chronic kidney disease and kidney failure. The age of symptom onset, progression and severity can vary greatly from one person to another, even among members of the same family. Some individuals may have mild cases that go undiagnosed well into adulthood; others may de ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Subcutaneous Administration
Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion. A subcutaneous injection is administered as a bolus into the subcutis, the layer of skin directly below the dermis and epidermis, collectively referred to as the cutis. The instruments are usually a hypodermic needle and a syringe. Subcutaneous injections are highly effective in administering medications such as insulin, morphine, diacetylmorphine and goserelin. Subcutaneous administration may be abbreviated as SC, SQ, subcu, sub-Q, SubQ, or subcut. Subcut is the preferred abbreviation to reduce the risk of misunderstanding and potential errors. Subcutaneous tissue has few blood vessels and so drugs injected into it are intended for slow, sustained rates of absorption, often with some amount of depot effect. Compared with other routes of administration, it is slower than intramuscular injections but still faster than intradermal injections. Subcutaneous infusion (as ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Medication
Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmacotherapy) is an important part of the medicine, medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management. Drugs are Drug class, classified in many ways. One of the key divisions is by level of controlled substance, control, which distinguishes prescription drugs (those that a pharmacist dispenses only on the medical prescription) from over-the-counter drugs (those that consumers can order for themselves). Medicines may be classified by mode of action, route of administration, biological system affected, or therapeutic effects. The World Health Organization keeps a list of essential medicines. Drug discovery and drug development are complex and expensive endeavors undertake ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lactate Dehydrogenase A
Lactate dehydrogenase A (LDHA) is an enzyme which in humans is encoded by the ''LDHA'' gene. It is a monomer of lactate dehydrogenase, which exists as a tetramer. The other main subunit is lactate dehydrogenase B (LDHB). Function Lactate dehydrogenase A catalyzes the inter-conversion of pyruvate and L- lactate with concomitant inter-conversion of NADH and NAD+. LDHA is found in most somatic tissues, though predominantly in muscle tissue and tumors, and belongs to the lactate dehydrogenase family. It has long been known that many human cancers have higher LDHA levels compared to normal tissues. It has also been shown that LDHA plays an important role in the development, invasion and metastasis of malignancies. Mutations in LDHA have been linked to exertional myoglobinuria. Interactive pathway map LDHA Inhibitors The following compounds have been demonstrated to inhibit the LDHA enzyme: * Oxamate * Epigallocatechin gallate * Quinoline 3-sulfonamide * CHK-336 CHK-336 is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Small Interfering RNA
Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA, double-stranded non-coding RNA, non-coding RNA, RNA molecules, typically 20–24 base pairs in length, similar to microRNA (miRNA), and operating within the RNA interference (RNAi) pathway. It interferes with the gene expression, expression of specific genes with complementary nucleotide sequences by degrading messenger RNA (mRNA) after Transcription (biology), transcription, preventing translation (biology), translation. Text was copied from this source, which is available under Creative Commons Attribution 4.0 International License It was discovered in 1998 by Andrew Fire at the Carnegie Institution for Science in Washington, D.C. and Craig Mello at the University of Massachusetts in Worcester. Structure Naturally occurring siRNAs have a well-defined structure that is a short (usually 20 to 24-base pair, bp) double-stranded RNA (dsRNA) with phosph ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
First-in-class Medication
A first-in-class medication is a prototype drug 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 medications and reports on them annually, first-in-class is not considered a regulatory category. Although many first-in-class medications qualify as breakthrough therapies, Regenerative Medicine Advanced Therapies and/or orphan drugs, first-in-class status itself has no regulatory effect. Examples Controversy Safety By definition, a first-in-class drug does not have the safety evidence from analogous products that not-first-in-class drugs would have. However, a study investigating recalls and warnings in relation to first-in-class drugs approved between 1997 and 2012 by Health Canada has found that first-in-class drugs actually have a more favourable benefit-to-harm ratio. Economics First-in-class drugs are often seen as commerc ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits. In the United States, indications for prescription drugs are approved by the FDA. Indications are included in the Indications and Usage section of the Prescribing Information. The primary role of this section of labeling is to enable health care practitioners to readily identify appropriate therapies for patients by clearly communicating the drug's approved indication(s). The Indications and Usage section states the disease or condition, or manifestation or symptoms thereof ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oxalate
Oxalate (systematic IUPAC name: ethanedioate) is an anion with the chemical formula . This dianion is colorless. It occurs naturally, including in some foods. It forms a variety of salts, for example sodium oxalate (), and several esters such as dimethyl oxalate (). It is a conjugate base of oxalic acid. At neutral pH in aqueous solution, oxalic acid converts completely to oxalate. Relationship to oxalic acid The dissociation of protons from oxalic acid proceeds in a determined order; as for other polyprotic acids, loss of a single proton results in the monovalent hydrogenoxalate anion . A salt with this anion is sometimes called an acid oxalate, monobasic oxalate, or hydrogen oxalate. The equilibrium constant ( ''K''a) for loss of the first proton is ( p''K''a = 1.27). The loss of the second proton, which yields the oxalate ion, has an equilibrium constant of (p''K''a = 4.28). These values imply, in solutions with neutral pH, no oxalic acid and only t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Breakthrough Therapy
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "breakthrough therapy" designation is not intended to imply that a drug is actually a "breakthrough" or that there is high-quality evidence of treatment efficacy for a particular condition; rather, it allows the FDA to grant priority review to drug candidates if preliminary clinical trials indicate that the therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases. The FDA has other mechanisms for expediting the review and approval process for promising drugs, including fast track designation, accelerated approval, and priority review. Requirements A breakthrough therapy designation can be assigned to a drug if "it is a drug which is intended alone or in combination wit ... [...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] |
