princessjdc
New member
I read on cff.org that Ataluren is in the 3 phase and it is testing to see if it helps to improve the lung function of those with a non-sense mutation.
Here is what it says, directly from the site.
Link
<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/treatments/Pipeline/
">http://www.cff.org/treatments/Pipeline/
</a>
Ataluren (formerly known as PTC124): PTC Therapeutics - A novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It aims to treat CF patients who have what is known as a "nonsense mutation." It has been demonstrated to be safe, orally available and well tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results. The Phase 3 trial began in Summer 2009, which is a 48-week study of ataluren in people with CF age 6 and older. The main goal of the trial is to find out if ataluren can improve lung function in people with the disease.
Here is what it says, directly from the site.
Link
<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/treatments/Pipeline/
">http://www.cff.org/treatments/Pipeline/
</a>
Ataluren (formerly known as PTC124): PTC Therapeutics - A novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It aims to treat CF patients who have what is known as a "nonsense mutation." It has been demonstrated to be safe, orally available and well tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results. The Phase 3 trial began in Summer 2009, which is a 48-week study of ataluren in people with CF age 6 and older. The main goal of the trial is to find out if ataluren can improve lung function in people with the disease.