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Casimersen
go.drugbank.com/drugs/DB14984ApprovedInvestigational[A218171, A218176, A229988] The application of antisense oligonucleotides in DMD patients with specific mutations allows for exon skipping to produce truncated BMD-like dystrophin proteins, which restore … A related, albeit a less severe, form of muscular dystrophy known as Becker muscular dystrophy (BMD) is characterized by shortened and partially functional dystrophin protein production.
Categories: Compounds used in a research, industrial, or household settingAlteplase
go.drugbank.com/drugs/DB00009ApprovedInvestigational[L43125] It cleaves plasminogen to form plasmin, an enzyme involved in the degradation of fibrin clots. … [A252345,L43125] It was first approved by the FDA in 1987 for the management of thromboembolic disease, including acute myocardial infarction (AMI).
Synonyms: t-PA, t-plasminogen activatorPivmecillinam
go.drugbank.com/drugs/DB01605ApprovedInvestigationalPivmecillinam is a mecillinam prodrug, a pivaloyloxymethyl ester of amdinocillin that is well absorbed orally, but broken down to amdinocillin in the intestinal mucosa. … It is active against gram-negative organisms and used as for amdinocillin. [PubChem]
Categories: Antibacterials for Systemic Use, Antiinfectives for Systemic UsePolysilicone-15
go.drugbank.com/drugs/DB11271ApprovedThe compound is a colorless to pale yellow viscous liquid which is soluble in organic solvents of medium polarity and insoluble in water [L2796, L2797, L2799, F87, F88, F89]. … It is the first polymeric UVB filer consisting of chromophores attached to a silicone backbone [L2796, L2797, L2799, F87, F88, F89].
Voxelotor
go.drugbank.com/drugs/DB14975Approved[T734] Voxelotor was granted accelerated FDA approval on November 25 2019, as it is likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition … to 20 million others worldwide.
Modafinil
go.drugbank.com/drugs/DB00745ApprovedInvestigationalThe exact mechanism of action is unclear, although in vitro studies have shown it to inhibit the reuptake of dopamine by binding to the dopamine reuptake pump, and lead to an increase in extracellular … Narcolepsy is caused by dysfunction of a family of wakefulness-promoting and sleep-suppressing peptides, the orexins, whose neurons are activated by modafinil.
Categories: Psychostimulants, Agents Used for ADHD and NootropicsDidanosine
go.drugbank.com/drugs/DB00900ApprovedInvestigationalDidanosine is a potent inhibitor of HIV replication, acting as a chain-terminator of viral DNA by binding to reverse transcriptase; ddI is then metabolized to dideoxyadenosine triphosphate, its putative … This modification prevents the formation of phosphodiester linkages which are needed for the completion of nucleic acid chains.
Categories: Antivirals for Systemic Use, Antiinfectives for Systemic UseEdoxaban
go.drugbank.com/drugs/DB09075ApprovedInvestigationalBy inhibiting factor Xa, a key protein in the coagulation cascade, edoxaban prevents the stepwise amplification of protein factors needed to form blood clots. … Traditionally, warfarin, a vitamin K antagonist, was used for stroke prevention in these individuals but effective use of this drug is limited by it's delayed onset, narrow therapeutic window, need for
Bacillus calmette-guerin substrain russian BCG-I live antigen
go.drugbank.com/drugs/DB16648ApprovedInvestigationalIt was developed to alleviate Canada's multi-year shortage of BCG vaccine, and is expected to be available by mid-2021.[L32734] … thought to work by stimulating a local immune and inflammatory response in the bladder.
Dexmethylphenidate
go.drugbank.com/drugs/DB06701ApprovedInvestigationalThe d-isomer is thought to have greater effect with fewer side effects than the l-isomer or the racemic mixture[A177181]. … It is used for treatment of Attention Deficit Hyperactivity Disorder (ADHD)[Label,A177181].
Categories: Psychostimulants, Agents Used for ADHD and Nootropics, Serotonergic Drugs Shown to Increase Risk of Serotonin Syndrome