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Cedazuridine
go.drugbank.com/drugs/DB15694ApprovedInvestigational[A215107, A215112, A215117, A215127, L14897] Cedazuridine was first reported in 2014,[A215107] and was subsequently approved by the FDA on July 7, 2020, in combination with [decitabine] for sale by … Myelodysplastic syndromes (MDS) are a group of hematopoietic neoplasms that give rise to variable cytopenias progressing to secondary acute myeloid leukemia (sAML), which is invariably fatal if untreated
Synonyms: (4R)-2'-Deoxy-2',2'-difluoro-3,4,5,6-tetrahydrouridineCategories: Heterocyclic Compounds, 1-RingPegvaliase
go.drugbank.com/drugs/DB12839ApprovedInvestigationalThe primary goal of lifelong treatment of PKU, as recommended by the American College of Medical Genetics and Genomics (ACMG) guidelines, is to maintain blood Phe concentration in the range of 120 µmol … Phenylketonuria is a rare autosomal recessive disorder that is characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH) [A33284] and affects about 1 in 10,000 to 15,000 people in the United
Plerixafor
go.drugbank.com/drugs/DB06809ApprovedInvestigationalSince CXCR4 and SDF-1α are involved in the trafficking and homing of CD34+ cells to the marrow compartment, blocking this interaction leads to an increase in CD34+ cell circulating levels. … [A7117,L45678] It is used to stimulate the release of stem cells from the bone marrow into the blood in patients with non-Hodgkin's lymphoma (NHL) and multiple myeloma to stimulate their immune system.
Products: MOZOBIL®, Enjeksiyonluk Çözelti, 1 ADETFerrous ascorbate
go.drugbank.com/drugs/DB14490ApprovedWithdrawnSynonyms: Iron(2+) L-ascorbate, L-Ascorbic acid, iron complexCategories: Compounds used in a research, industrial, or household settingMetreleptin
go.drugbank.com/drugs/DB09046ApprovedInvestigationalMetreleptin, a recombinant analog of the human hormone leptin, is an orphan drug used to treat complications of leptin deficiency in people with lipodystrophy. … [L41315] In February 2014, metreleptin was approved by the FDA for the treatment of complications of leptin deficiency, as an adjunct to diet, in patients with congenital generalized or acquired generalized
Synonyms: N-Methionylleptin, r-metHuLeptinApolizumab
go.drugbank.com/drugs/DB06467InvestigationalApolizumab is a humanized monoclonal antibody that is being studied as a treatment for hematologic cancers.
Synonyms: IMMUNOGLOBULIN G1, ANTI-(HUMAN HISTOCOMPATIBILITY ANTIGEN HLA-DR) (HUMAN-MOUSE MONOCLONAL HU1D10.GAMMA.(SUB 1)-CHAIN), DISULPHIDE WITH HUMAN-MOUSE MONOCLONAL HU1D10 LIGHT CHAIN, DIMERDeutetrabenazine
go.drugbank.com/drugs/DB12161ApprovedInvestigationalIn clinical trials of patients with HD, 12 weeks of treatment of deutetrabenazine resulted in overall improvement in mean total maximal chorea scores and motor signs than placebo [A32046]. … This allows less frequent dosing and a lower daily dose with improvement in tolerability [A32043].
Synonyms: D6 tetrabenazine, D6-tetrabenazineCategories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP1A2 SubstratesDigitoxin
go.drugbank.com/drugs/DB01396ApprovedWithdrawnA cardiac glycoside sometimes used in place of digoxin. It has a longer half-life than digoxin; toxic effects, which are similar to those of digoxin, are longer lasting.
Categories: Compounds used in a research, industrial, or household setting, P-glycoprotein substrates with a Narrow Therapeutic IndexFencamfamin
go.drugbank.com/drugs/DB01463ApprovedIllicitWithdrawnFencamfamin (Glucoenergan, Reactivan) is a stimulant which was developed in the 1960s as an appetite suppressant. … Fencamfamin is used for treating depressive day-time fatigue, lack of concentration and lethargy. It is especially useful in patients with chronic conditions due to its favourable safety profile.
Pegunigalsidase alfa
go.drugbank.com/drugs/DB14992ApprovedInvestigationalPegunigalsidase alfa (PRX-102) is a recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease, a rare genetic disorder characterized … [A259347,A259352] In May 2023, the EMA granted marketing authorization to pegunigalsidase alfa in the European Union (EU) for the treatment of adult patients with Fabry disease.