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Navepegritide
go.drugbank.com/drugs/DB17824ApprovedInvestigational[L56065] Navepegritide was approved by the US FDA in February 2026 for use in patients 2 years of age and older with achondroplasia with open epiphyses.[L56058,L56065] … [L56058,A275435] Navepegritide is a prodrug of active C-type natriuretic peptide (CNP) consisting of a CNP moiety transiently conjugated to two methoxy polyethylene glycol (mPEG) moieties via a proprietary
Cystathionine beta-Synthase Deficiency
smpdb.ca/view/SMP0000177DiseaseCystathionine Beta-Synthase Deficiency (CBS Deficiency; Homocystinuria) is an autosomal recessive disease caused by a mutation in the CBS gene which codes for cystathionine beta-synthase. A deficiency in this enzyme results in accumulati...
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseMethionine Adenosyltransferase Deficiency
smpdb.ca/view/SMP0000221DiseaseMethionine adenosyltransferase (MAT; Hypermethioninemia; MAT I/III deficiency) deficiency is caused by mutations in the MAT1A gene which causes isolated hypermethioninemia. MAT catalyzes the formation of adenosylmethionine from methionin...
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseHypermethioninemia
smpdb.ca/view/SMP0000341DiseaseThis gene is responsible for Adenosylhomocysteinase, an enzyme which takes S-adenosyl homocysteine as input, and produces homocysteine as its output.
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseMethionine Metabolism
smpdb.ca/view/SMP0000033Metabolicin a methylated product plus S-adenosylhomocysteine, and the conversion of S-adenosylhomocysteine to produce the compounds homocysteine and adenosine. … These pathways have three common reactions with both pathways including the transformation of methionine to S-adenosylmethionine (SAM), the use of SAM in many different transmethylation reactions resulting
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseMethylenetetrahydrofolate Reductase Deficiency (MTHFRD)
smpdb.ca/view/SMP0000340DiseaseMethylenetetrahydrofolate reductase deficiency (MTHFRD; Homocystinuria due to defect of n(5,10)-methylene THF deficiency) is caused by a defect in the MTHFR gene which codes for methylenetetrahydrofolate reductase. Methylenetetrahydrofol...
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseHomocystinuria-Megaloblastic Anemia Due to Defect in Cobalamin Metabolism, cblG Complementation Type
smpdb.ca/view/SMP0125684DiseaseHomocystinuria-megaloblastic anemia due to defect in cobalamin metabolism, cblG complementation type, also known as methionine synthase deficiency or methylcobalamin deficiency, cblG type, is a rare inborn error of metabolism (IEM) and a...
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseMigalastat
go.drugbank.com/drugs/DB05018ApprovedInvestigational[L47036,L47057] As of August 2018, migalastat under Amicus Therapeutics' brand name Galafold is currently approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland, and … Fabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A).
Categories: Alpha-Galactosidase A (alpha-Gal A) Pharmacological Chaperones, Heterocyclic Compounds, 1-RingSynonyms: (2R,3S,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol, 1-DeoxygalactostatinGlycine N-Methyltransferase Deficiency
smpdb.ca/view/SMP0000222DiseaseGNMT catalyzes the conversion of glycine into N-methylglycine (sarcosine) using S-adenosylmethionine (SAM or AdoMet).
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylaseHomocystinuria-Megaloblastic Anemia Due to Defect in Cobalamin Metabolism, cblG Complementation Type
smpdb.ca/view/SMP0000570DiseaseHomocystinuria-megaloblastic anemia due to defect in cobalamin metabolism, cblG complementation type, also known as methionine synthase deficiency or methylcobalamin deficiency, cblG type, is a rare inborn error of metabolism (IEM) and a...
Drugs: Pyridoxal phosphate · Tetrahydrofolic acid · Ademetionine · Choline · Adenosine phosphate · Serine +17 moreEnzymes: S-adenosylmethionine decarboxylase proenzyme, S-methyl-5'-thioadenosine phosphorylase