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Lonafarnib
go.drugbank.com/drugs/DB06448ApprovedInvestigationalHutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant disorder estimated to affect approximately one in 20 million individuals resulting in adverse symptoms associated with premature … aberrant farnesylated form of lamin A called progerin in the inner nuclear membrane.
Cobimetinib
go.drugbank.com/drugs/DB05239ApprovedInvestigationalIt has been approved in Switzerland and the US, in combination with vemurafenib for the treatment of patients with unresectable or metastatic BRAF V600 mutation-positive melanoma. … Cobimetinib is an orally active, potent and highly selective small molecule inhibiting mitogen-activated protein kinase kinase 1 (MAP2K1 or MEK1), and central components of the RAS/RAF/MEK/ERK signal transduction
Diethylcarbamazine
go.drugbank.com/drugs/DB00711ApprovedInvestigationalVet approvedWithdrawnAn anthelmintic used primarily as the citrate in the treatment of filariasis, particularly infestations with Wucheria bancrofti or Loa loa.
Propyliodone
go.drugbank.com/drugs/DB09366ApprovedRadiopaque medium usually in oil; used in bronchography.
Categories: Compounds used in a research, industrial, or household settingDeoxycholic acid
go.drugbank.com/drugs/DB03619ApprovedInvestigationalDeoxycholic acid is a a bile acid which emulsifies and solubilizes dietary fats in the intestine, and when injected subcutaneously, it disrupts cell membranes in adipocytes and destroys fat cells in that … In April 2015, deoxycholic acid was approved by the FDA for the treatment submental fat to improve aesthetic appearance and reduce facial fullness or convexity.
Paltusotine
go.drugbank.com/drugs/DB16277ApprovedInvestigational[A274476] Paltusotine was approved by the FDA on September 25, 2025 for the treatment of acromegaly in adults.[L54061]
Ubrogepant
go.drugbank.com/drugs/DB15328ApprovedInvestigational[erenumab], [fremanezumab], [galcanezumab]) have also been approved in recent years.[A189207] Ubrogepant was approved by Health Canada on November 10, 2022. … Ubrogepant is indicated for the acute treatment of migraine headaches with or without aura in adults.
Amlexanox
go.drugbank.com/drugs/DB01025ApprovedWithdrawnAmlexanox is an antiallergic drug, clinically effective for atopic diseases, especially allergic asthma and rhinitis. … Recurrent aphthous ulcer (RAU) is the most prevalent oral mucosal disease in humans, estimated to affect between 5% and 50% of the general population.
Arimoclomol
go.drugbank.com/drugs/DB05025Approved[L51384] Arimoclomol was approved by the FDA on September 20, 2024, making it the first treatment for Niemann-Pick disease, type C (NPC). … [L51389] It is also being investigated in amyotrophic lateral sclerosis (ALS) [A264414, A264429] and inclusion body myositis.[A264419]
Synonyms: 3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY), 1-OXIDE, N-[(2R)-2-Hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride, 1-oxideObecabtagene autoleucel
go.drugbank.com/drugs/DB17362ApprovedInvestigational[A265040,A265045] It works by targeting B-cell-specific CD19 surface markers present on malignant B cells. … [L52450] Obecabtagene autoleucel was also investigated in other hematological cancers and systemic lupus erythematosus.[A265045]
Synonyms: AUTOLOGOUS CD4+ AND CD8+ T CELLS, ENRICHED FROM PERIPHERAL BLOOD MONONUCLEAR CELLS (PBMCS) OBTAINED BY APHERESIS TRANSDUCED EX VIVO WITH A NON-REPLICATING, SELF-INACTIVATING (SIN) LENTIVIRAL VECTOR, ENCODING