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Vosoritide
go.drugbank.com/drugs/DB11928ApprovedInvestigationalAchondroplasia is an autosomal dominant genetic disease and the most common cause of dwarfism in humans. … [A242277] It results from a gain-of-function missense mutation in _FGFR3_ that results in a dramatic suppression of bone growth, both in volume and in length.
Cholestyramine
go.drugbank.com/drugs/DB01432ApprovedInvestigationalCholestyramine resin is quite hydrophilic, but insoluble in water. … Cholestyramine or colestyramine is a bile acid sequestrant. Bile acid sequestrants are polymeric compounds which serve as ion exchange resins.
Categories: Compounds used in a research, industrial, or household setting, Ion Exchange ResinsProducts: ANTILIP GRANULADO EN SACHET, PHL-cholestyramine Light - 210g TinOnasemnogene abeparvovec
go.drugbank.com/drugs/DB15528ApprovedInvestigationalOnasemnogene abeparvovec is the first gene therapy that was approved for this indication in the USA. … [Nusinersen] is another gene therapy that is currently approved by the FDA for the treatment of SMA in pediatric and adult patients.
Categories: Genetic TherapySertaconazole
go.drugbank.com/drugs/DB01153ApprovedSertaconazole nitrate is an antifungal medication of the imidazole class. It is available in topical formulations for the treatment of skin infections such as athlete's foot.
RAPA-501-Allo
go.drugbank.com/drugs/DB15890InvestigationalRAPA-501-ALLO is composed of T-cells extracted from healthy volunteers; these cells are then reprogrammed _ex vivo_ in a process that involves de-differentiation and re-differentiation.[L16483]
Acylcarnitine Dec-4-enedioylcarnitine
smpdb.ca/view/SMP0123874MetabolicOnce in the matrix, dec-4-enedioylcarnitine can react with the carnitine O-palmitoyltransferase 2 enzyme found in the mitochondrial inner membrane to once again form dec-4-enedioyl-CoA and L-carnitine. … In forming an acylcarnitine derivative, dec-4-enedioyl-CoA reacts with L-carnitine to form dec-4-enedioylcarnitine. This reaction is catalyzed by carnitine O-palmitoyltransferase.
Drugs: Biotin · Adenosine phosphate · ATP · Levocarnitine · Magnesium cation · Pyrophosphoric acid +1 moreAcylcarnitine Dec-3-enedioylcarnitine
smpdb.ca/view/SMP0123877MetabolicOnce in the matrix, dec-3-enedioylcarnitine can react with the carnitine O-palmitoyltransferase 2 enzyme found in the mitochondrial inner membrane to once again form dec-3-enedioyl-CoA and L-carnitine. … In forming an acylcarnitine derivative, dec-3-enedioyl-CoA reacts with L-carnitine to form dec-3-enedioylcarnitine. This reaction is catalyzed by carnitine O-palmitoyltransferase.
Drugs: Biotin · Adenosine phosphate · ATP · Levocarnitine · Magnesium cation · Pyrophosphoric acid +1 moreCeritinib
go.drugbank.com/drugs/DB09063ApprovedInvestigationalThe FDA approved ceritinib in April 2014 due to a surprisingly high response rate (56%) towards crizotinib-resistant tumours and has designated it with orphan drug status. … Following treatment with crizotinib (a first-generation ALK inhibitor), most tumours develop drug resistance due to mutations in key "gatekeeper" residues of the enzyme.
Synonyms: N-{2-[(5-chloro-2-{[2-isopropoxy-5-methyl-4-(piperidin-4-yl)phenyl]amino}pyrimidin-4-yl)amino]phenyl}propane-2-sulfonamideAcylcarnitine Dec-5-enedioylcarnitine
smpdb.ca/view/SMP0123875MetabolicOnce in the matrix, dec-5-enedioylcarnitine can react with the carnitine O-palmitoyltransferase 2 enzyme found in the mitochondrial inner membrane to once again form dec-5-enedioyl-CoA and L-carnitine. … In forming an acylcarnitine derivative, dec-5-enedioyl-CoA reacts with L-carnitine to form dec-5-enedioylcarnitine. This reaction is catalyzed by carnitine O-palmitoyltransferase.
Drugs: Biotin · Adenosine phosphate · ATP · Levocarnitine · Magnesium cation · Pyrophosphoric acid +1 morePulmonary Tuberculosis resistant to isoniazid, rifamycins, a fluoroquinolone and a second line injectable antibacterial drug
go.drugbank.com/conditions/DBCOND0154352Drugs: PretomanidSynonyms: Pulmonary Tuberculosis resistant to isoniazid, rifamycins, a fluoroquinolone and a second line injectable antibacterial drug