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Doxecitine
go.drugbank.com/drugs/DB02594ApprovedInvestigationalIt is an essential component of the deoxyribonucleotide pool required for DNA synthesis and repair. … This combination is the first and only approved treatment for Thymidine Kinase 2 deficiency.[L54613, L54618]
Stiripentol
go.drugbank.com/drugs/DB09118ApprovedInvestigational[A250825] However, its clinical efficacy as adjunctive therapy for epilepsies stems from its inhibitory action on CYP enzymes, as stiripentol reduces the degradation of CYP-sensitive antiepileptic drugs … [A19740, A250825] The clinical development and marketing of stiripentol were first delayed due to the drug's potent inhibitory effects on hepatic cytochrome P450 (CYP) enzymes.
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). … Arimoclomol is a hydroxylamine derivative [A264414] and a heat-shock protein-70 (HSP70) co-inducer.
Sodium Depletion
go.drugbank.com/conditions/DBCOND0093130Drugs: Sodium chlorideSynonyms: Low salt syndrome, Syndrome low saltBenzgalantamine
go.drugbank.com/drugs/DB19353ApprovedInvestigational[L51564] Benzgalantamine was approved by the FDA in July 2024 for the treatment of mild-to-moderate dementia in Alzheimer's patients.[L51534,L51564] … Gastrointestinal adverse effects are the most frequently reported side effects in patients undergoing treatment with cholinesterase inhibitors, including galantamine, and are often a reason for treatment
Deflazacort
go.drugbank.com/drugs/DB11921Approved[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. … [L6694] Duchenne Muscular Dystrophy is an inherited disorder resulting from mutations of the dystrophin gene, which is important for muscle function.
Categories: Corticosteroids for Systemic Use, Corticosteroids for Systemic Use, PlainTapinarof
go.drugbank.com/drugs/DB06083ApprovedInvestigationalIt is available as a topical cream to be applied to the affected area once daily. … Tapinarof is a novel, first-in-class, small-molecule AhR agonist that is indicated for the treatment of adult psoriasis.
Categories: Antipsoriatics for Topical UseCeritinib
go.drugbank.com/drugs/DB09063ApprovedInvestigationalCeritinib is used for the treatment of adults with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) following failure (secondary to resistance or intolerance) of … This occurrence led to development of novel second-generation ALK inhibitors such as ceritinib to overcome crizotinib resistance.
Iptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigational[A262581] On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. … [A262586] Additionally, iptacopan has the benefit of targeting factor B, which only affect the alternative complement pathway, leaving the classic and lectin pathway untouched for the body to still mount
Categories: Compounds used in a research, industrial, or household settingOlaparib
go.drugbank.com/drugs/DB09074ApprovedInvestigational[A246015] Olaparib is used to treat a number of BRCA-associated tumours, including ovarian cancer, breast cancer, pancreatic cancer, and prostate cancer. … [L41100, L40908, L43792] It was first approved by the FDA and EU in December 2014,[A246020] and by Health Canada in April 2016.[L42820]