I received this email from PTC pharmaceutical wanted to share this news (this my gene type G542X
Top-Line Analysis from the Ataluren Phase 3 Trial in Nonsense Mutation Cystic Fibrosis to be Presented at the European Cystic Fibrosis Conference
South Plainfield, NJ - June 7, 2012 - Results from a Phase 3 clinical study of ataluren in nonsense mutation cystic fibrosis (nmCF) will be presented at the European Cystic Fibrosis Conference in Dublin, Ireland. Michael Konstan, M.D., a principal investigator for PTC Therapeutics' Phase 3 trial at University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio will present top-line data on the efficacy and safety of ataluren in nmCF during Workshop 22 - Late Breaking Science at 5:00 pm (17:00) on Friday, June 8th.
Two additional presentations focusing on trial data will also be presented at the conference. Michael Wilschanski, M.D., a principal investigator for the trial at Hadassah University Hospital in Jerusalem, Israel, will present baseline data from the study in Symposium 24, Enhancing CFTR Functions - What's in the Pipeline? on Friday, June 8 at 10:30 am. Christiane De Boeck, M.D., a principal investigator for the trial at University Hospital Leuven will highlight the ataluren clinical study in the plenary session, Potentiating and Correcting CFTR at 11:00 am on Saturday, June 9th.
The conference is taking place at the Convention Centre Dublin from June 6-9th.
About Ataluren
An investigational new drug discovered by PTC Therapeutics, ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as CFTR in nmCF. The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
For more information on PTC Therapeutics, please visit the company's web site at www.ptcbio.com.
Top-Line Analysis from the Ataluren Phase 3 Trial in Nonsense Mutation Cystic Fibrosis to be Presented at the European Cystic Fibrosis Conference
South Plainfield, NJ - June 7, 2012 - Results from a Phase 3 clinical study of ataluren in nonsense mutation cystic fibrosis (nmCF) will be presented at the European Cystic Fibrosis Conference in Dublin, Ireland. Michael Konstan, M.D., a principal investigator for PTC Therapeutics' Phase 3 trial at University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio will present top-line data on the efficacy and safety of ataluren in nmCF during Workshop 22 - Late Breaking Science at 5:00 pm (17:00) on Friday, June 8th.
Two additional presentations focusing on trial data will also be presented at the conference. Michael Wilschanski, M.D., a principal investigator for the trial at Hadassah University Hospital in Jerusalem, Israel, will present baseline data from the study in Symposium 24, Enhancing CFTR Functions - What's in the Pipeline? on Friday, June 8 at 10:30 am. Christiane De Boeck, M.D., a principal investigator for the trial at University Hospital Leuven will highlight the ataluren clinical study in the plenary session, Potentiating and Correcting CFTR at 11:00 am on Saturday, June 9th.
The conference is taking place at the Convention Centre Dublin from June 6-9th.
About Ataluren
An investigational new drug discovered by PTC Therapeutics, ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as CFTR in nmCF. The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
For more information on PTC Therapeutics, please visit the company's web site at www.ptcbio.com.