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Cysteamine
go.drugbank.com/drugs/DB00847ApprovedInvestigational[L15611] On August 25th 2020, the first ophthalmic solution for cystinosis requiring only 4 daily treatments was granted FDA approval.
Bucetin
go.drugbank.com/drugs/DB13278ApprovedWithdrawnBucetin is an analgesic and antipyretic medication which was approved for use in Germany but was withdrawn from the market in 1986 due to renal toxicity caused by the medication.[A175636]
Deflazacort
go.drugbank.com/drugs/DB11921ApprovedDeflazacort, also known as Emflaza, is a corticosteroid prodrug used as an agent to manage Duchenne Muscular Dystrophy (DMD). … [A179446,A179449,L6697] This disease usually manifests by muscle weakness in early childhood followed by loss of the ability to walk (ambulation) as early as age 7.
Navitoclax
go.drugbank.com/drugs/DB12340InvestigationalNavitoclax has been used in trials studying the treatment and basic science of Solid Tumors, Non-Hodgkin's Lymphoma, EGFR Activating Mutation, Chronic Lymphoid Leukemia, and Hematological Malignancies,
Cycloserine
go.drugbank.com/drugs/DB00260ApprovedInvestigationalAntibiotic substance produced by Streptomyces garyphalus.
Siponimod
go.drugbank.com/drugs/DB12371ApprovedInvestigationalIt was approved by the FDA on March 26, 2019 [L5792] and by Health Canada on February 20, 2020. … Siponimod, also known as _Mayzent_, by Novartis, is a new drug formulated for the management of Multiple Sclerosis (MS).
GEM-231
go.drugbank.com/drugs/DB05798InvestigationalGEM231 is a second-generation antisense oligonucleotide targeting the mRNA of the R1alpha regulatory subunit of cAMP dependent protein kinase. A monoclonal antibody combined with a toxic substance that is used treat certain types of acut...
Daleuton
go.drugbank.com/drugs/DB00154InvestigationalNutraceuticalA 20-carbon-chain fatty acid, unsaturated at positions 8, 11, and 14. It differs from arachidonic acid, 5,8,11,14-eicosatetraenoic acid, only at position 5.
Synthetic Conjugated Estrogens, A
go.drugbank.com/drugs/DB09317ApprovedAvailable as either "Synthetic Conjugated Estrogens, A" containing 9 estrogen derivatives (available as Cenestin) or as "Synthetic Conjugated Estrogens, B" containing 10 estrogen derivatives (available … as Enjuvia), these products are isolated as precursors from yam or soy plants and then chemically modified to mimic the products available in their naturally occurring form.
Deutivacaftor
go.drugbank.com/drugs/DB15141ApprovedInvestigational[L52275,A179677] Deutivacaftor was first approved by the US FDA in December 2024 in combination with two CFTR correctors - [tezacaftor] and [vanzacaftor] - for the treatment of patients with responsive