Emma Mueller, Author at The Boomer Esiason Foundation

March 19, 2019

SOURCE: CFF.org Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), an affiliate of the Cystic Fibrosis Foundation, announced it will extend funding for continued collaboration with Proteostasis Therapeutics, Inc., for the development of new therapies to treat the most common CF mutation, Delta F508. Proteostasis will continue to focus...

U.S. FDA Approves KALYDECO™ for Use in Eight Additional CF Mutations

by Emma Mueller

CF News, Research, Therapies

March 19, 2019

SOURCE - VERTEX PRESS RELEASE KALYDECO is the first medicine to treat the underlying cause of CF for people with specific mutations in the CFTR gene KALYDECO facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein The eight additional mutations are present in...

Scientists Pinpoint 105 Additional Genetic Errors that Cause CF

by Emma Mueller

CF News, Research

March 19, 2019

SOURCE: CF Roundtable Of the over 1,900 errors already reported in the gene responsible for cystic fibrosis (CF), it is unclear how many of them actually contribute to the inherited disease. Now a team of researchers reports significant headway in figuring out which mutations are benign...

Phase 2 Study of Kalydeco and VX-661 Shows Promising Increases in Lung Function

by Emma Mueller

CF News, Research

March 19, 2019

Vertex Pharmaceuticals, Inc., maker of the cystic fibrosis drug Kalydeco, has announced the promising results of a phase 2 study it has been conducting on the combination of Kalydeco and VX-661. Patients with two copies of the F508del mutation, after taking the combination, showed an...

Vertex Announces Initiation of a Phase 3 Study for CF Patients with the F508del Mutation

by Emma Mueller

CF News, Research

March 19, 2019

On February 26, 2013, Vertex Pharmaceuticals, Inc. announced the initiation of a Phase 3 development program for people with cystic fibrosis who have two copies of the F508del mutation. The program will study various fixed-dose combinations of lumacaftor (VX-809) and ivacaftor over two 24-week Phase...

NHLBI has Launched a Program on Early Cystic Fibrosis Lung Disease

by Emma Mueller

CF News, Research

March 19, 2019

The NHLBI has launched a program where researchers will study pre-symptomatic lung disease in infants and young children who have cystic fibrosis (CF), results of which could show exactly how CF develops. These results could provide further insight into the origin and progession of the...

FDA Advisory Committee supports the use of Novartis inhalation powder, Tobramycin

by Emma Mueller

Boomer Esiason Foundation, CF Community, CF News, Research, Therapies

March 19, 2019

The FDA Advisory Committee voted 13 to 1 that research showed adequate evidence to support the use of tobramycin inhalation powder by cystic fibrosis patients whose lungs contain the bacteria Pseudomonas aeruginosa, the leading cause of lung function loss in patients with CF. Tobramycin inhalation...

Gene Network Restores Cystic Fibrosis Protein Function

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

Source: Science Daily (July 31, 2012) Cystic fibrosis is an inherited disease caused by mutations in a gene that adversely affect its protein product. In its correct form and cellular location, this protein, cystic fibrosis transmembrane conductance regulator (CFTR), functions as a channel for ions to...

Final Results from Phase 2 Combination Study of Kalydeco and VX-809 Show Significant Improvements

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

Source: Cystic Fibrosis Foundation A Phase 2 clinical trial of Kalydeco™ in combination with VX-809 showed significant improvements in lung function in people with the most common CF mutation, according to final results announced today by Vertex Pharmaceuticals Inc. Both Kalydeco and VX-809, a CF drug in...

Concentrated Saline Therapy Not Effective in Young Children with CF, Study Finds

by Emma Mueller

CF News, Research, Therapies

March 19, 2019

Inhaling concentrated saline (salt water) mist does not reduce how often infants and young children with cystic fibrosis need antibiotics for respiratory symptoms, according to findings from a clinical trial sponsored by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes...

Author: Emma Mueller

CFF Therapeutics Extends Drug Development Collaboration via CFF.org

U.S. FDA Approves KALYDECO™ for Use in Eight Additional CF Mutations

Scientists Pinpoint 105 Additional Genetic Errors that Cause CF

Phase 2 Study of Kalydeco and VX-661 Shows Promising Increases in Lung Function

Vertex Announces Initiation of a Phase 3 Study for CF Patients with the F508del Mutation

NHLBI has Launched a Program on Early Cystic Fibrosis Lung Disease

FDA Advisory Committee supports the use of Novartis inhalation powder, Tobramycin

Gene Network Restores Cystic Fibrosis Protein Function

Final Results from Phase 2 Combination Study of Kalydeco and VX-809 Show Significant Improvements

Concentrated Saline Therapy Not Effective in Young Children with CF, Study Finds

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