U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS
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Showing 1 - 10 of 18 results

Status:
US Approved Rx (2024)
First approved in 2024

Class (Stereo):
CHEMICAL (ACHIRAL)



Gavinostat is an orally bioavailable hydroxymate inhibitor of histone deacetylase (HDAC) with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. Gavinostat inhibits class I and class II HDACs, resulting in an accumulation of...
Status:
Investigational

Class (Stereo):
CHEMICAL (ACHIRAL)

Targets:


Ezutromid (SMTC-1100) is a small molecule utrophin upregulator. Ezutromid was identified from an iterative analoging approach from initial hits identified using a human muscle-specific utrophin A promoter cell-based assay. It increases both utrophin ...
Status:
Investigational

Class (Stereo):
CHEMICAL (ABSOLUTE)



GLPG-0492, an orally available selective androgen receptor modulator (SARM) was tested in a Phase I Proof of Mechanism study to assess the effect on muscle function in healthy volunteers. A biomarker effect similar to that of Oxandrolone was observed...
Status:
Investigational

Class (Stereo):
CHEMICAL (RACEMIC)



Halofuginone is a low molecular weight quinazolinone alkaloid, and a potent inhibitor of collagen alpha1(I) and matrix metalloproteinase 2 (MMP-2) gene expression. Halofuginone also effectively suppresses tumor progression and metastasis in mice. Hal...
Status:
US Previously Marketed
First approved in 1973

Class (Stereo):
CHEMICAL (RACEMIC)


Mazindol is an amphetamine-like medicine which was developed by Sandoz in 1967 and approved by FDA for the treatment of obesity and Duchenne muscular dystrophy under the names Sanorex and Mazanor. The exact mechanism of action is unknown, but possib...
Status:
Possibly Marketed Outside US
First approved in 2012

Class (Stereo):
CHEMICAL (ABSOLUTE)



Epigallocatechin-3-gallate (EGCG), the major polyphenol from green tea, has the potential to impact a variety of human diseases. EGCG functions as a powerful antioxidant, preventing oxidative damage in healthy cells, but also as an antiangiogenic and...
Status:
Possibly Marketed Outside US
First approved in 2011

Class (Stereo):
CHEMICAL (ACHIRAL)



Idebenone is a synthetic short-chain benzoquinone and a substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport and supplementing cellular energy levels. Idebenone was initially developed...
Status:
Possibly Marketed Outside US

Class (Stereo):
CHEMICAL (ABSOLUTE)



Arbekacin is a broad-spectrum aminoglycoside used to treat methicillin-resistant Staphylococcus aureus (MRSA). Arbekacin has antibacterial activities against high-level gentamicin-resistant Enterococci, multidrug-resistant Pseudomonas aeruginosa, and...
Status:
Possibly Marketed Outside US

Class (Stereo):
CHEMICAL (ACHIRAL)

Targets:


Ataluren (Translarna) is a small-molecule drug approved in Europe for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation. Ataluren interacts with the ribosome enabling it to read through premature nonsense stop signals on mRNA...
Status:
Investigational

Class:
PROTEIN



Angiotensin (1-7) [Ang 1-7] is a 7 amino acid peptide generated predominantly from Ang II by the action of Ang-converting enzyme 2. Ang 1-7 can act as a negative modulator of aldosterone secretion in vitro and in vivo. The endogenous heptapeptide ang...

Showing 1 - 10 of 18 results