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Fosdenopterin
go.drugbank.com/drugs/DB16628Approved[A230088] MoCD is incurable and median survival in untreated patients is approximately 36 months[A230088] - treatment, then, is focused on improving survival and maintaining neurological function. … [L43075] In September 2022, the EMA approved the use of fosdenopterin.[L43372,L43433]
Trioxsalen
go.drugbank.com/drugs/DB04571ApprovedWithdrawnThe photoactivated form produces interstrand linkages in DNA resulting in cell apoptosis. … It is administered either topically or orally in conjunction with UV-A (the least damaging form of ultraviolet light) for phototherapy treatment of vitiligo and hand eczema.
ALGRX 1207
go.drugbank.com/drugs/DB06482ExperimentalALGRX 1207 entered clinical trials for cutaneous neuropathic pain, such as chemotherapy-induced neuropathy, in 2006. It was being developed by Anesiva, but trials have halted. … ALGRX 1207 is a topical local anesthetic that acts by binding to the fast sodium channel.
Phenylacetic acid
go.drugbank.com/drugs/DB09269ApprovedBecause it is used in the illicit production of phenylacetone (used in the manufacture of substituted amphetamines), it is subject to controls in countries including the United States and China. … Phenylacetic acid is an organic compound containing a phenyl functional group and a carboxylic acid functional group. It is a white solid with a disagreeable odor.
Categories: Ammonium Ion Binding ActivityEplontersen
go.drugbank.com/drugs/DB16199ApprovedInvestigationalThis approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS … [A262975] Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of insoluble and misfolded amyloid in
Synonyms: Ion-ttr-lrxRegdanvimab
go.drugbank.com/drugs/DB16405ApprovedWithdrawn[A235830,A241995] It received its first full approval in South Korea in September 2021,[A241995] followed by the EU in November 2021.[L39130] … Trials investigating the use of regdanvimab as a therapeutic candidate for the treatment of COVID-19 began in mid-2020.
Stamulumab
go.drugbank.com/drugs/DB05915InvestigationalMYO-029 is a human anti-GDF-8 monoclonal antibody, which is being developed to treat muscle-wasting diseases including muscular dystrophy and age-related sarcopenia.
Brexpiprazole
go.drugbank.com/drugs/DB09128ApprovedInvestigational[A182186] Currently approved for the treatment of depression, schizophrenia, and agitation associated with dementia due to Alzheimer’s disease, brexpiprazole has also been investigated in other psychiatric … [L46417] Although it is structurally similar to [aripiprazole], brexpiprazole has different binding affinities for dopamine and serotonin receptors.
Dimethyltryptamine
go.drugbank.com/drugs/DB01488IllicitInvestigationalAn N-methylated indoleamine derivative, a serotonergic hallucinogen found in several plants, especially Prestonia amazonica (Apocynaceae) and in mammalian brain, blood, and urine. … It apparently acts as an agonist at some types of serotonin receptors and an antagonist at others.
Synonyms: N,N-dimethyltryptamine, N,N-dimethyl-1H-indole-3-ethylaminePegcrisantaspase
go.drugbank.com/drugs/DB16281InvestigationalPegcrisantaspase is under investigation in clinical trial NCT02257684 (A Dose Confirmation and Pharmacokinetic Study of Pegcrisantaspase Administered as Intravenous (IV) Infusion in Children and Young … Adults With Acute Lymphoblastic Leukemia (ALL) /Lymphoblastic Lymphoma (LBL).