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Blue Diaper Syndrome
smpdb.ca/view/SMP0000583DiseaseBlue diaper syndrome is a recessive metabolic disorder that has not yet been determined to be X-linked or autosomal. This syndrome is caused by a mutation in the large neutral amino acids transporter small subunit 1 protein, which allows...
Enzymes: Y+L amino acid transporter 2, Y+L amino acid transporter 1Lysinuric Protein Intolerance (LPI)
smpdb.ca/view/SMP0000585DiseaseIt is caused by a mutation in the SLC7A7 gene which encodes the Y+L amino acid transporter 1 protein, which is involved in the uptake of amino acids, both with sodium for neutral amino acids, and without
Enzymes: Y+L amino acid transporter 2, Y+L amino acid transporter 1Paltusotine
go.drugbank.com/drugs/DB16277ApprovedInvestigational[A274476] Paltusotine was approved by the FDA on September 25, 2025 for the treatment of acromegaly in adults.[L54061] … hormone (GH) and decreases the production of growth-promoting insulin-like growth factor 1 (IGF-1).
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2C19 InhibitorsFolitixorin calcium
go.drugbank.com/drugs/DB05308InvestigationalANX-510 (CoFactor) is a folate-based biomodulator drug being developed to enhance the activity and reduce associated toxicity of the widely used cancer chemotherapy, 5-fluorouracil (5-FU). … CoFactor use with 5-FU is being evaluated in clinical trials in first line treatment of metastatic colorectal cancer.
Phenformin
go.drugbank.com/drugs/DB00914ApprovedWithdrawn(From Martindale, The Extra Pharmacopoeia, 30th ed, p290) … A biguanide hypoglycemic agent with actions and uses similar to those of metformin.
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2D6 SubstratesSynonyms: β-PhenethybiguanideErythrityl tetranitrate
go.drugbank.com/drugs/DB01613ApprovedWithdrawnA vasodilator with general properties similar to nitroglycerin. (From Martindale, The Extra Pharmacopoeia, 30th ed, p1020)
Afamitresgene autoleucel
go.drugbank.com/drugs/DB18592ApprovedInvestigationalAfamitresgene autoleucel is a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T cell immunotherapy product consisting of CD4 and CD8 positive T cells transduced with a … [L51114] The transduced T cells are expanded, washed, formulated into a suspension, and cryopreserved.
Synonyms: AUTOLOGOUS CD4+ AND CD8+ T CELLS OBTAINED BY LEUKAPHERESIS, TRANSDUCED WITH AN HIV-DERIVED SELF-INACTIVATING (SIN) LENTIVIRAL VECTOR, ENCODING AN ENHANCED-AFFINITY T CELL RECEPTOR (TCR) SPECIFIC FOR THE, HUMAN MELANOMA ANTIGEN A4 (MAGE FAMILY MEMBER A4, APrednisolone sodium metazoate
go.drugbank.com/drugs/DB05340InvestigationalATL-2502(Colal-Pred) is an old drug (prednisolone metasulphobenzoate, a steroid) with a new delivery system that releases the medication only in the colon.
Mixtures: KORTISETIN GÖZ POMADI, 5 G, KORTISETIN GÖZ POMADI, 2.5 GPumactant
go.drugbank.com/drugs/DB04955ExperimentalIt is a unique formulation of the naturally occurring phospholipids Dipalmitoylphosphatidycholine (DPPC) and Phosphatidyl Glycerol (PG). … Pumactant is a synthetic surfactant used for treating respiratory distress syndrome in neonates.
Nipocalimab
go.drugbank.com/drugs/DB16257ApprovedInvestigational[L64042] It is an aglycosylated, fully human IgG1 monoclonal antibody that binds FcRn and blocks the receptor's recycling of immunoglobulin G (IgG), thereby lowering circulating IgG levels, including pathogenic … [L64042] It is administered by intravenous infusion.[L64042] Nipocalimab-aahu was first approved by the FDA in April 2025 for generalized myasthenia gravis.
Synonyms: .-1 chain), disulfide with human monoclonal m281 .lambda.-chain, dimer, FCGRT (Fc fragment of IgG receptor and transporter, neonatal Fc receptor, FcRn, transmembrane alpha chain of the neonatal receptor)], Homo sapiens monoclonal antibody (gamma1 heavy chain Homo sapiens (1-