Ataluren(CAS: 775304-57-9)

Ataluren, also known as PTC124, is a chemical compound used in the treatment of Duchenne muscular dystrophy due to a nonsense mutation. Its chemical formula is C13H17N3O3. Ataluren is a non-nucleoside trans-acting factor that binds to the nonsense codons in mRNA, allowing for the translation of the full-length protein.

Ataluren is primarily used in the treatment of Duchenne muscular dystrophy (DMD) associated with a nonsense mutation. It helps in the production of full-length dystrophin protein by binding to premature stop codons in the mRNA, thereby promoting the production of functional dystrophin.

Ataluren has been evaluated for safety in clinical trials. It is generally well-tolerated, with the most common side effects being headache, dizziness, and nausea. However, it is important to follow safety guidelines, including conducting regular health check-ups and monitoring for any adverse reactions. Always store the compound in a secure location, away from children and pets.

Ataluren should be stored in a cool, dry place, away from direct sunlight and moisture. It is recommended to keep the compound in its original container to protect it from contamination. Ensure that the storage area is well-ventilated and maintain a temperature between 15°C and 25°C. Always check the expiration date and discard any unused portions that have passed the expiration date.