Add like
Add dislike
Add to saved papers

Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.

PTC-124, a 1,2,4-oxadiazole compound, is in development by PTC Therapeutics Inc as an orally active small molecule that can override nonsense stop translation signals to produce full-length proteins. PTC-124 is currently being evaluated in phase II clinical trials against cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD). The functional properties of PTC-124 are similar to the aminoglycoside antibiotic gentamicin, but the two compounds are chemically distinct and PTC-124 does not exhibit any antibiotic characteristics. In vitro experiments showed PTC-124 to be superior to gentamicin at ribosomal read-through of nonsense mutations. In vivo investigations revealed that PTC-124 was effective in restoring the production of full-length protein in animal models of CF and DMD. Phase I clinical trials reported that PTC-124 was well tolerated in healthy patients. The author concludes that the encouraging results observed to date make PTC-124 an attractive option for further well-designed, long-term human studies on larger sample populations. The author also predicts that if results continue to be positive, PTC-124 could also be trialed in other single gene disorders with nonsense mutations such as hemophilia, neurofibromatosis, retinitis pigmentosa, bullous skin diseases and lysosomal storage disorders.

Full text links

We have located links that may give you full text access.
Can't access the paper?
Try logging in through your university/institutional subscription. For a smoother one-click institutional access experience, please use our mobile app.

Related Resources

For the best experience, use the Read mobile app

Mobile app image

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app

All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC.
This website also contains material copyrighted by 3rd parties.

By using this service, you agree to our terms of use and privacy policy.

Your Privacy Choices Toggle icon

You can now claim free CME credits for this literature searchClaim now

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app