CAS 775304-57-9
:Ataluren
Description:
Ataluren, with the CAS number 775304-57-9, is a small molecule drug primarily used in the treatment of genetic disorders caused by nonsense mutations, particularly in Duchenne muscular dystrophy (DMD). It functions as a read-through agent, promoting the ribosomal incorporation of amino acids at premature stop codons, thereby allowing for the production of full-length functional proteins. Ataluren is characterized by its ability to selectively enhance the translation of mRNA that contains these mutations, which can lead to improved muscle function in affected individuals. The compound is typically administered orally and has been evaluated for its safety and efficacy in clinical trials. Its pharmacokinetics involve absorption, distribution, metabolism, and excretion processes that are crucial for its therapeutic action. While Ataluren has shown promise in clinical settings, its use may be accompanied by side effects, and its effectiveness can vary among patients. Overall, Ataluren represents a significant advancement in the treatment of genetic conditions linked to nonsense mutations, highlighting the potential of targeted therapies in modern medicine.
Formula:C15H9FN2O3
InChI:InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
InChI key:InChIKey=OOUGLTULBSNHNF-UHFFFAOYSA-N
SMILES:FC1=C(C2=NC(=NO2)C3=CC(C(O)=O)=CC=C3)C=CC=C1
Synonyms:- 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoicacid
- Atalure
- Ataluren
- Benzoic acid,3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-
- Pct 124
- Ptc 124
- Translarna
- 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
- Ataluren(PTC124)
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Found 8 products.
3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid
CAS:Purity:95.0%Color and Shape:SolidMolecular weight:284.2460021972656Ataluren
CAS:<p>Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations.</p>Formula:C15H9FN2O3Purity:99.16% - 99.84%Color and Shape:SolidMolecular weight:284.243-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid
CAS:<p>3-(5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid</p>Formula:C15H9FN2O3Purity:98%Color and Shape: white powderMolecular weight:284.24g/molPTC-124
CAS:Controlled Product<p>Applications PTC-124 is a nonaminoglycoside that has been reported to induce ribosomes to read through premature nonsense stop signals on mRNA, allowing the production of full-length functional proteins,<br>References Yu, H. et al.: Int. J. Mol. Med., 33, 729 (2014); Aditya, S. et al.: J. Biomed. Pharm. Res., 2, 21 (2013);<br></p>Formula:C15H9FN2O3Color and Shape:White To Off-WhiteMolecular weight:284.24Ataluren
CAS:<p>Ataluren is a drug that was developed to treat Duchenne muscular dystrophy. It is an orally administered small molecule that has been shown to restore the production of a protein called dystrophin, which is missing in people with Duchenne muscular dystrophy. The drug works by binding to the RNA polymerase and terminating transcription of the mutated gene that causes the disease. Ataluren has been studied in clinical trials as an alternative to steroids, which are used to slow or stop muscle deterioration and allow the body's natural healing process to take place. This drug has also been shown to be effective against other mutations, including those associated with cystic fibrosis and Huntington's disease.</p>Formula:C15H9FN2O3Purity:Min. 95%Color and Shape:PowderMolecular weight:284.24 g/mol






