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Displaying drugs 701 - 725 of 15485 in total
Avalglucosidase alfa, or NeoGAA, is a drug for enzyme replacement therapy specifically designed for Pompe disease, a rare inherited neuromuscular disorder caused by the deficiency of the alpha-glucosidase (GAA) enzyme. GAA is an essential enzyme that hydrolyzes glycogen into free glucose for use in cellular functions. In Pompe disease, the...
Approved
Investigational
Matched Description: … , especially in smooth, cardiac, and skeletal muscle cells. ... muscle weakness, which leads to premature death and debilitating effects on people’s lives. ... In Pompe disease, the GAA enzyme is missing and patients are unable to properly break down glycogen, …
Matched Categories: … Enzymes and Coenzymes ... Alimentary Tract and Metabolism …
Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). The expression of hFVIII-SQ is driven by a liver-specific promoter, which enables hepatocytes to produce factor VIII protein and increase the levels of active...
Approved
Investigational
Matched Description: … [L43282,A252807] Valoctocogene roxaparvovec was approved by EMA in September 2022 and is indicated for ... hFVIII-SQ is driven by a liver-specific promoter, which enables hepatocytes to produce factor VIII protein and ... A252797] As opposed to these therapies, valoctocogene roxaparvovec offers the advantage of continuous and
Matched Categories: … Cellular and Gene Therapy ... Blood and Blood Forming Organs ... Amino Acids, Peptides, and Proteins …
Mantle cell lymphoma is a heterogeneous sub-category of non-Hodgkin's lymphoma that can be classified as either an aggressive nodal or an indolent leukemic non-nodal variant. Despite the introduction of Bruton's tyrosine kinase (BTK) inhibitors such as ibrutinib and acalabrutinib, the prognosis for MCL patients remains poor and those that relapse...
Approved
Matched Description: … ], the prognosis for MCL patients remains poor and those that relapse following BTK inhibitor therapy ... Despite the introduction of Bruton's tyrosine kinase (BTK) inhibitors such as [ibrutinib] and [acalabrutinib ... therapies have been developed that modify a patient's own T cells using viral transduction to bind to and
Matched Categories: … Amino Acids, Peptides, and Proteins ... Antineoplastic cell and gene therapy ... Receptors, Cytoplasmic and Nuclear ... Antineoplastic and Immunomodulating Agents …
Guselkumab is a human immunoglobulin G1 lambda (IgG1λ) monoclonal antibody that selectively blocks interleukin-23. IL-23 is an inflammatory cytokine that activates the CD4+ T-helper (Th17) cell pathway to mediate the inflammatory cascade that induces psoriatic plaque formation . In clinical trials, guselkumab demonstrated improved skin clearance and symptomatic improvements in...
Approved
Investigational
Matched Description: … In clinical trials, guselkumab demonstrated improved skin clearance and symptomatic improvements in dermatological …
Matched Categories: … Amino Acids, Peptides, and Proteins ... Skin and Mucous Membrane Agents ... Antineoplastic and Immunomodulating Agents …
Pegunigalsidase alfa (PRX-102) is a recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease, a rare genetic disorder characterized by the deficiency of alpha-galactosidase A.[A259352,L46342] Unlike other forms of recombinant alpha-galactosidase A, such as agalsidase alfa and agalsidase beta, pegunigalsidase alfa uses a...
Approved
Investigational
Matched Description: … allowing it to reach target organs with a lower dose and frequency of administration. ... [A259352,L46342] Unlike other forms of recombinant alpha-galactosidase A, such as [agalsidase alfa] and ... While agalsidase alfa and agalsidase beta enter lysosomes via the mannose-6-phosphate (M6P) receptor, …
Matched Categories: … Enzymes and Coenzymes ... Alimentary Tract and Metabolism …
A potent lipoxygenase inhibitor that interferes with arachidonic acid metabolism. The compound also inhibits formyltetrahydrofolate synthetase, carboxylesterase, and cyclooxygenase to a lesser extent. It also serves as an antioxidant in fats and oils. [PubChem]
Approved
Investigational
Matched Description: … It also serves as an antioxidant in fats and oils. [PubChem] ... The compound also inhibits formyltetrahydrofolate synthetase, carboxylesterase, and cyclooxygenase to …
Matched Categories: … Antineoplastic and Immunomodulating Agents …
Trilostane is an inhibitor of 3 beta-hydroxysteroid dehydrogenase used in the treatment of Cushing's syndrome. It was withdrawn from the United States market in April 1994.
Approved
Investigational
Vet approved
Withdrawn
Matched Categories: … Sex Hormones and Insulins ... Hormones, Hormone Substitutes, and Hormone Antagonists …
Acetylcarnitine is an investigational drug in the United states, Italy, United Kingdom, China, Israel, and Norway, and it is approved in Italy, Portugal, Argentina, Chile, Philippines, Australia, and India. Acetylcarnitine can be synthesized, but it is also naturally found in adequate amounts in healthy humans. In human plasma and tissues,...
Approved
Investigational
Matched Description: … Norway, and it is approved in Italy, Portugal, Argentina, Chile, Philippines, Australia, and India. ... is an amino acid derivative that is made in the kidney, liver, and brain from lysine and methionine. ... Acetylcarnitine is an investigational drug in the United states, Italy, United Kingdom, China, Israel, and
Matched Categories: … Psychostimulants, Agents Used for ADHD and Nootropics …
Glycerol phenylbutyrate is a nitrogen-binding agent. Chemically, it is a triglyceride in which three molecules of phenylbutyrate are linked to a glycerol backbone. FDA approved on February 1, 2013.
Approved
Matched Categories: … Alimentary Tract and Metabolism ... Various Alimentary Tract and Metabolism Products …
Approved
Investigational
Matched Categories: … Sex Hormones and Insulins …
Technetium Tc-99m (99mTc) succimer, also known as technetium Tc-99m dimercaptosuccinic acid (DMSA), is a radioactive diagnostic agent used to evaluate functional renal tissue and tubular function in patients with renal parenchymal disorders. It works by binding to the cortical region of the kidneys. Technetium Tc-99m succimer is used with gamma...
Approved
Matched Description: … dimercaptosuccinic acid (DMSA),[A245643] is a radioactive diagnostic agent used to evaluate functional renal tissue and
Matched Categories: … Indicators and Reagents …
Gaxilose has been used in trials studying the diagnostic of Lactose Intolerance.
Approved
Investigational
Lactitol, also known as 4-β-D-galactopyranosyl-D-glucitol, is a sugar alcohol synthesized from lactose. It is used in food manufacturing as a nutritive sweetener and is approximately 35% as sweet as table sugar (i.e. sucrose). Clinically, lactitol has been investigated for use as an osmotic laxative and, along with other non-absorbable disaccharides...
Approved
Investigational
Matched Description: … [A190918] It is used in food manufacturing as a nutritive sweetener and is approximately 35% as sweet ... [A190912] Clinically, lactitol has been investigated for use as an osmotic laxative and, along with other …
Matched Categories: … Diet, Food, and Nutrition ... Alimentary Tract and Metabolism …
Estradiol Cypionate is a pro-drug ester of DB00783, a naturally occurring hormone that circulates endogenously within the human body. Estradiol is the most potent form of all mammalian estrogenic steroids and acts as the major female sex hormone. As a pro-drug of estradiol, estradiol cypionate therefore has the same downstream...
Approved
Investigational
Vet approved
Matched Iupac: … (1S,3aS,3bR,9bS,11aS)-7-hydroxy-11a-methyl-1H,2H,3H,3aH,3bH,4H,5H,9bH,10H,11H,11aH-cyclopenta[a]phenanthren …
Matched Description: … Because of the difference in potency between estradiol and estrone, menopause (and a change in primary ... skin, prostate, bone, fat, and brain. ... a chance to enter systemic circulation and exert its estrogenic effects [A12102]. …
Matched Categories: … Hormones, Hormone Substitutes, and Hormone Antagonists …
Approved
Experimental
Flunisolide (marketed as AeroBid, Nasalide, Nasarel) is a corticosteroid with anti-inflammatory actions. It is often prescribed as treatment for allergic rhinitis and its principle mechanism of action involves activation of glucocorticoid receptors.
Approved
Investigational
Matched Description: … It is often prescribed as treatment for allergic rhinitis and its principle mechanism of action involves …
A corticosteroid used topically in the treatment of various skin disorders. It is usually employed as a cream or an ointment, and is also used as a polyethylene tape with an adhesive. (From Martindale, The Extra Pharmacopoeia, 30th ed, p733)
Approved
Matched Description: … It is usually employed as a cream or an ointment, and is also used as a polyethylene tape with an adhesive …
Matched Categories: … fludroxycortide and antibiotics ... Hormones, Hormone Substitutes, and Hormone Antagonists …
A nonfluorinated corticosteroid anti-inflammatory agent used topically for dermatoses.
Approved
Investigational
Matched Categories: … desonide and antiseptics …
Ethanolamine oleate is a mild sclerosing agent. It is composed of ethanolamine, a basic substance, which when combined with oleic acid forms a clear, straw to pale yellow colored, deliquescent oleate.
Approved
Sodium aurothiomalate is a gold compound that is used for its immunosuppressive anti-rheumatic effects. Gold Sodium Thiomalate is supplied as a solution for intramuscular injection containing 50 mg of Gold Sodium Thiomalate per mL. It is most effective in active progressive rheumatoid arthritis and of little or no value in...
Approved
Investigational
Matched Description: … It is most effective in active progressive rheumatoid arthritis and of little or no value in the presence …
Matched Categories: … Antiinflammatory and Antirheumatic Products …
Testosterone undecanoate is the ester prodrug of testosterone and has a mid-chain fatty acid at the carbon 17β position. It was developed via fatty acid esterification of testosterone in order to achieve orally administer testosterone. There are oral and intramuscular formulations available for testosterone undecanoate: both formulations are indicated for...
Approved
Investigational
Matched Description: … of testosterone deficiency, such as decreased libido, erectile dysfunction, and loss of muscle and bone ... Testosterone undecanoate is the ester prodrug of [testosterone] and has a mid-chain fatty acid at the ... development of the male sex organs and for the maintenance of secondary sex characteristics. …
Matched Categories: … Testosterone and derivatives ... Hormones, Hormone Substitutes, and Hormone Antagonists …
Ixekizumab is a humanized immunoglobulin G subclass 4 (IgG4) monoclonal antibody (mAb) against interleukin-17A (IL-17A) and prevents it from interacting with the IL-17A receptor. As IL-17A is a pro-inflammatory cytokine involved in inflammation and immune responses, blocking its effect is beneficial for use in inflammatory conditions. In particular, IL-17A has...
Approved
Investigational
Matched Description: … humanized immunoglobulin G subclass 4 (IgG4) monoclonal antibody (mAb) against interleukin-17A (IL-17A) and ... As IL-17A is a pro-inflammatory cytokine involved in inflammation and immune responses, blocking its ... has been found to be implicated in a variety of autoimmune diseases including Rheumatoid Arthritis and
Matched Categories: … Amino Acids, Peptides, and Proteins ... Skin and Mucous Membrane Agents ... Antineoplastic and Immunomodulating Agents …
Lenograstim is a recombinant granulocyte colony-stimulating factor used as an immunostimulating agent.
Approved
Investigational
Matched Categories: … Amino Acids, Peptides, and Proteins ... Antineoplastic and Immunomodulating Agents ... Intercellular Signaling Peptides and Proteins …
Collagenase clostridium histolyticum is an enzyme produced by the bacterium Clostridium histolyticum. It is beneficial in the breakdown of collagen plaques for the treatment of Dupuytren's contracture and Peyronie's disease. The topical formulation is used for the debridement of necrotic tissue due to burns or chronic ulcers. On July 6,...
Approved
Investigational
Matched Description: … It is beneficial in the breakdown of collagen plaques for the treatment of Dupuytren's contracture and ... [L14872] Also known as Qwo, this injection is the first approved injectable treatment for cellulite and
Matched Categories: … Enzymes and Coenzymes ... Skin and Mucous Membrane Agents ... Preparations for Treatment of Wounds and Ulcers …
Plicamycin is an antineoplastic antibiotic produced by Streptomyces plicatus. It has been used in the treatment of testicular cancer, Paget's disease of bone, and, rarely, the management of hypercalcemia. The manufacturer discontinued plicamycin in 2000.
Approved
Investigational
Withdrawn
Matched Iupac: … (2S,4R,5S,6R)-4,5-dihydroxy-6-methyloxan-2-yl]oxy}-5-hydroxy-6-methyloxan-2-yl]oxy}-8,9-dihydroxy-7-methyl
Matched Description: … It has been used in the treatment of testicular cancer, Paget's disease of bone, and, rarely, the management …
Matched Categories: … Indicators and Reagents ... Calcium-Regulating Hormones and Agents ... Antineoplastic and Immunomodulating Agents ... Cytotoxic Antibiotics and Related Substances …
Displaying drugs 701 - 725 of 15485 in total