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Migalastat
go.drugbank.com/drugs/DB05018ApprovedInvestigationalThis enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other … [L47036,L47057,L4274,L4278] In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated
Betula pendula tar oil
go.drugbank.com/drugs/DB11361ApprovedNutraceuticalBetula pendula tar oil is an essential oil found in the buds of the plant with the same name. … [T219] The utilization of this product started during the second world war in the Sovietic Union.
Letermovir
go.drugbank.com/drugs/DB12070ApprovedInvestigationalLetermovir recieved approval from the FDA on November 8th, 2017 for use in prophylaxis of cytomegalovirus (CMV) infection in allogeneic hematopoietic stem cell transplant patients. … It is currently marketed under the brand name Prevymis.[L1021]
Lamotrigine
go.drugbank.com/drugs/DB00555ApprovedInvestigationalLamotrigine is an antiepileptic drug belonging in the phenyltriazine class. It is used in the treatment of both epilepsy and as a mood stabilizer in bipolar disorder. … Lamotrigine is the first medication since lithium granted Food and Drug Administration (FDA) approval for the maintenance treatment of bipolar type I. It is approved for use in more than 30 countries.
Palbociclib
go.drugbank.com/drugs/DB09073ApprovedInvestigationalPalbociclib is a piperazine pyridopyrimidine[A176792] that acts in the cell cycle machinery. … [L5867] It was originally FDA approved on March 2015 for the treatment of HR-positive, HER2-negative advanced or metastatic breast cancer and its indications were updated in April 2019 to include male
Istiratumab
go.drugbank.com/drugs/DB15363InvestigationalIstiratumab is under investigation in clinical trial NCT02399137 (A Phase 2 Study of MM-141 Plus Nab-paclitaxel and Gemcitabine in Front-line Metastatic Pancreatic Cancer).
Lobaplatin
go.drugbank.com/drugs/DB13049InvestigationalLobaplatin has been used in trials studying the treatment of Breast Cancer, Hepatocellular Carcinoma, and Lobaplatin and Gemcitabine in Combination, Second-line Treatments on Advanced Osteosarcoma.
Olipudase alfa
go.drugbank.com/drugs/DB12835ApprovedInvestigational[A251590] Olipudase alfa gained its first global approval in Japan on March 28, 2022. … [L42740] ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin
Viltolarsen
go.drugbank.com/drugs/DB15005ApprovedInvestigationalis contingent on further verification in confirmatory trials. … [A218176] Viltolarsen was granted accelerated FDA approval on August 12, 2020, based on data showing an increase in dystrophin levels in skeletal muscle of patients treated with viltolarsen; this approval
Spesolimab
go.drugbank.com/drugs/DB15626ApprovedInvestigationalin December 2022 for the treatment of flares in adult patients with generalized pustular psoriasis. … It is a humanized monoclonal immunoglobulin G1 antibody that was produced in Chinese hamster ovary (CHO) cells by recombinant DNA technology.