Emma Mueller, Author at The Boomer Esiason Foundation

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...

Pharmaxis Announces Positive Combined Phase 3 CF Trial Results

by Emma Mueller

CF News, Therapies

March 19, 2019

Pharmaceutical company Pharmaxis today announced significant results of pooled data from its two large scale six month Phase III trials of Bronchitol (inhaled mannitol) in people with cystic fibrosis. The combined results have been presented for the first time at the North American Cystic Fibrosis Conference...

Gilead’s Cayston Beats Tobramycin Inhalation Solution in Six-Month Head-to-Head Study

by Emma Mueller

CF News, Therapies

March 19, 2019

Gilead Sciences, Inc. announced that its head-to-head Phase III clinical trial of Cayston® (aztreonam for inhalation solution) versus tobramycin inhalation solution (TIS) in cystic fibrosis (CF) patients with Pseudomonas aeruginosa (P. aeruginosa) achieved its co-primary endpoint of superiority of Cayston for mean actual change in...

Analysis Shows TOBI Associated With Reduced Mortality in CF Patients With Common Lung Infection

by Emma Mueller

CF News, Therapies

March 19, 2019

A new analysis of data from more than 12,000 people with cystic fibrosis (CF) and Pseudomonas aeruginosa (Pa) lung infection in the Cystic Fibrosis Foundation's Patient Registry shows that use of TOBI® (Tobramycin Inhalation Solution, USP) was associated with a 21% reduction in mortality in...

Vertex Pharmaceuticals Clinical Trial to Evaluate Combo of VX-770 and VX-809 Targeting CFTR Protein

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Inc. today announced the initiation of a Phase 2a clinical trial that will evaluate multiple combinations of VX-770 and VX-809, investigational oral cystic fibrosis transmembrane conductance regulator (CFTR) modulators for the treatment of cystic fibrosis (CF). The trial is designed to evaluate the...

PTC Therapeutics Awarded $1.6 Million FDA Grant to Support Ongoing Phase 3 Study of Ataluren

by Emma Mueller

CF News, Therapies

March 19, 2019

PTC Therapeutics, Inc. (PTC) today announced a grant award of approximately $1.6 million from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OPD) to support an ongoing Phase 3 study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF). The...

New CF Drugs Bring Hope; More Survive Into Adulthood, But They Face New Challenges

by Emma Mueller

CF News, Therapies

March 19, 2019

Emily Schaller likes to travel as part of the cystic-fibrosis advocacy she does, but the daily regimen she uses to manage her disease weighs her down. “You have to bring three extra bags with meds and the vest,” she said. Schaller, 28, takes five or six pills...

Eurand Reports Further Positive Data from Pancreatic Insufficiency Drug Study

by Emma Mueller

CF News, Therapies

March 19, 2019

Eurand Pharmaceuticals, a subsidiary of specialty pharmaceutical company Eurand, has announced additional data from a post-hoc analysis of a Phase III clinical trial with Zenpep delayed-release capsules, an FDA-approved pancreatic enzyme product, or PEP, for the treatment of exocrine pancreatic insufficiency in patients with cystic...

Cincinnati Children’s Cited in US News Report As a ‘Best’ Hospital for CF Kids

by Emma Mueller

CF News, Therapies

March 19, 2019

A 'Best' Hospital for Cystic Fibrosis Kids Unhappy with its so-so record, Cincinnati Children's used tools of health reform to turn itself around Waiting to be ushered into an outpatient examination room at Cincinnati Children's Hospital Medical Center, Alicia Lang doesn't chatter on like most 18-year-old girls....

Author: Emma Mueller

Phase 2 Results Show Ataluren Restores Production of Functional CFTR Protein

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

Pharmaxis Announces Positive Combined Phase 3 CF Trial Results

Gilead’s Cayston Beats Tobramycin Inhalation Solution in Six-Month Head-to-Head Study

Analysis Shows TOBI Associated With Reduced Mortality in CF Patients With Common Lung Infection

Vertex Pharmaceuticals Clinical Trial to Evaluate Combo of VX-770 and VX-809 Targeting CFTR Protein

PTC Therapeutics Awarded $1.6 Million FDA Grant to Support Ongoing Phase 3 Study of Ataluren

New CF Drugs Bring Hope; More Survive Into Adulthood, But They Face New Challenges

Eurand Reports Further Positive Data from Pancreatic Insufficiency Drug Study

Cincinnati Children’s Cited in US News Report As a ‘Best’ Hospital for CF Kids

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