Comments on: What in the world? http://saltyandsweet.org/2009/08/24/what-in-the-world/ Daily life fighting Cystic Fibrosis and Cystic Fibrosis Related Diabetes. Thu, 08 Sep 2011 19:43:29 +0000 hourly 1 http://wordpress.org/?v=3.3.1 By: Pete http://saltyandsweet.org/2009/08/24/what-in-the-world/comment-page-1/#comment-773 Pete Wed, 05 May 2010 16:35:15 +0000 http://saltyandsweet.org/?p=494#comment-773 check out http://www.cysticfibrosis.org.au/ this is our national site for australia, has a research link on thehome page if ur still interested in what researh is happening in other countries. hope you find our aussie site interesting pete check out http://www.cysticfibrosis.org.au/
this is our national site for australia, has a research link on thehome page if ur still interested in what researh is happening in other countries.
hope you find our aussie site interesting
pete

]]> By: Nathan http://saltyandsweet.org/2009/08/24/what-in-the-world/comment-page-1/#comment-549 Nathan Thu, 27 Aug 2009 12:28:51 +0000 http://saltyandsweet.org/?p=494#comment-549 Well, not so great news on Miglustat. I've been checking for updates on the Miglustat clinical trial, and noticed this morning that the Bio-IT World story has changed. When the story was first posted July 22nd, the story said that the study had concluded and that Actelion was "currently reviewing the results". Now the story says, "After reviewing the results of the Phase IIa study, Actelion decided not to continue clinical development for the time being. But in light of the French team's results (the study of Miglustat in CF respiratory cells), Actelion has decided to conduct further preclinical evaluation of the drug in CF before making any final decisions". This is both disappointing and frustrating. There is no mention in the Bio-IT World story of an update to the original posting. After digging around more on the Actelion website, I found in the press release, "Actelion announces Half Year financial results 2009" the same line, "The recently concluded Phase IIa proof-of-concept study does not support further clinical development at this time...". This seems to be the only information they give. They do not include any information about the clinical trial data that leads them to this decision. I will look into this more and send along any interesting information I find. I apologize for getting anyone's hopes up over this trial. As someone with CF, I was very hopeful myself that this would be a new and revolutionary treatment option. I am still hopeful that someone (maybe Vertex, maybe someone else) will develop a successful CFTR corrector that will be that next big step in CF treatment. Well, not so great news on Miglustat. I’ve been checking for updates on the Miglustat clinical trial, and noticed this morning that the Bio-IT World story has changed. When the story was first posted July 22nd, the story said that the study had concluded and that Actelion was “currently reviewing the results”. Now the story says, “After reviewing the results of the Phase IIa study, Actelion decided not to continue clinical development for the time being. But in light of the French team’s results (the study of Miglustat in CF respiratory cells), Actelion has decided to conduct further preclinical evaluation of the drug in CF before making any final decisions”.

This is both disappointing and frustrating. There is no mention in the Bio-IT World story of an update to the original posting. After digging around more on the Actelion website, I found in the press release, “Actelion announces Half Year financial results 2009″ the same line, “The recently concluded Phase IIa proof-of-concept study does not support further clinical development at this time…”. This seems to be the only information they give. They do not include any information about the clinical trial data that leads them to this decision. I will look into this more and send along any interesting information I find.

I apologize for getting anyone’s hopes up over this trial. As someone with CF, I was very hopeful myself that this would be a new and revolutionary treatment option. I am still hopeful that someone (maybe Vertex, maybe someone else) will develop a successful CFTR corrector that will be that next big step in CF treatment.

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