SI
SI
discoversearch

We've detected that you're using an ad content blocking browser plug-in or feature. Ads provide a critical source of revenue to the continued operation of Silicon Investor.  We ask that you disable ad blocking while on Silicon Investor in the best interests of our community.  If you are not using an ad blocker but are still receiving this message, make sure your browser's tracking protection is set to the 'standard' level.
Biotech / Medical : Biotech News
 Public ReplyPrvt ReplyMark as Last ReadFilePrevious 10Next 10PreviousNext  
To: tnsaf who wrote (6314)8/23/2008 3:16:39 AM
From: idos   of 7143
 
PTC124 - this technology holds the potential applicability to the suppression of nonsense mutations and its MoA has been validated in animals and clinical trials, showed efficacy signals (treatment resulted in the partial restoration of the protein encoded by the mutant gene), and was found to be safe. However, it will only work where the cause of the disease is a nonsense mutation. Patients not bearing the nonsense mutation (90% of CF patients for example) are going to need other therapies.
Report TOU ViolationShare This Post
 Public ReplyPrvt ReplyMark as Last ReadFilePrevious 10Next 10PreviousNext  

HomeMailHotSubjectMarksPeopleMarksKeepersSettings
Terms Of UseContact UsCopyright/IP PolicyPrivacy PolicyAbout UsFAQAdvertise on SI
© 2025 Knight Sac Media.  Data provided by Twelve Data, Alpha Vantage, and CityFALCON News