Emma Mueller, Author at The Boomer Esiason Foundation

March 19, 2019

Vertex Pharmaceuticals and the Cystic Fibrosis Foundation today announced positive results from an ongoing Phase 3 clinical trial of VX-770 for children age 6 to 11. VX-770 is an oral drug in development that targets the underlying cause of cystic fibrosis. Children who were on the drug...

VX-770 Achieves Positive Results in Phase 3 Clinical Trial, Vertex Announces

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Incorporated today announced positive results from the Phase 3 STRIVE study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). STRIVE was designed to evaluate people with a mutation in the CF gene known as G551D. In...

FDA Committee Declines to Recommend Liprotamase for EPI

by Emma Mueller

CF News, Therapies

March 19, 2019

The FDA’s Gastrointestinal Drugs Advisory Committee (GDAC) has voted to recommend non-approval of Eli Lilly’s Solpura (liprotamase), a non-porcine pancreatic enzyme replacement therapy, currently under Agency review for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis and pancreatectomy. During the meeting, the GDAC...

Mpex Starts Phase 3 Clinical Trial of Aeroquin? for Treating Chronic Bacterial Infections in CF

by Emma Mueller

CF News, Therapies

March 19, 2019

Mpex Pharmaceuticals, Inc., today during the JP Morgan 29th Annual Healthcare Conference, announced that it has initiated its Phase 3 clinical trial program with Aeroquin™ (MP-376) for the treatment of pulmonary infections in patients with cystic fibrosis. Aeroquin is Mpex's proprietary aerosol formulation of levofloxacin,...

Inspire Reports Disappointing Results for TIGER-2 Denufosol Phase 3 Clinical Trial

by Emma Mueller

CF News, Therapies

March 19, 2019

Inspire Pharmaceuticals, Inc. announced today the top-line results from its second Phase 3 clinical trial, TIGER-2, with denufosol tetrasodium for the treatment of cystic fibrosis. The trial did not achieve statistical significance for its primary efficacy endpoint, which was change from baseline in FEV1 (Forced Expiratory...

Phase 3 Trial of Ataluren Expected to be Completed in 2011

by Emma Mueller

CF News, Therapies

March 19, 2019

PTC Therapeutics, Inc. announced today that it has completed enrollment of a Phase 3 clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). The 48-week study is designed to determine whether ataluren can improve lung function in patients with...

Denufosol Phase 3 Trial Published in Leading Medical Journal

by Emma Mueller

CF Community, CF News, Therapies

March 19, 2019

Inspire Pharmaceuticals, Inc. announced today that the results from its first Phase 3 clinical trial with denufosol tetrasodium for cystic fibrosis (CF), TIGER-1, have been published in the peer-reviewed publication, American Journal of Respiratory and Critical Care Medicine (AJRCCM). Denufosol is an investigational, inhaled, novel...

New England Journal of Medicine Publishes Phase 2 Study of VX-770 for Treating Cause of CF

by Emma Mueller

CF News, Therapies

March 19, 2019

In a study published in this week’s New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase 2 clinical trial of 39 people with cystic fibrosis. There were...

Phase 2 Results Show Ataluren Restores Production of Functional CFTR Protein

by Emma Mueller

CF News, Therapies

March 19, 2019

PTC Therapeutics, Inc. today announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine. The published data show that treatment with ataluren, an investigational...

Discovery Labs KL4 Surfactant Granted Orphan Drug Designation for the Treatment of CF

by Emma Mueller

CF News, Therapies

March 19, 2019

Discovery Laboratories, Inc. today announced that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to Discovery’s KL4 surfactant for the treatment of cystic fibrosis (CF). Orphan designation provides for up to seven years...

Author: Emma Mueller

VX-770 Trial: ‘Profound Improvement’ in Kids

VX-770 Achieves Positive Results in Phase 3 Clinical Trial, Vertex Announces

FDA Committee Declines to Recommend Liprotamase for EPI

Mpex Starts Phase 3 Clinical Trial of Aeroquin? for Treating Chronic Bacterial Infections in CF

Inspire Reports Disappointing Results for TIGER-2 Denufosol Phase 3 Clinical Trial

Phase 3 Trial of Ataluren Expected to be Completed in 2011

Denufosol Phase 3 Trial Published in Leading Medical Journal

New England Journal of Medicine Publishes Phase 2 Study of VX-770 for Treating Cause of CF

Phase 2 Results Show Ataluren Restores Production of Functional CFTR Protein

Discovery Labs KL4 Surfactant Granted Orphan Drug Designation for the Treatment of CF

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