BEF, Author at The Boomer Esiason Foundation

March 19, 2019

SOURCE [Posted by Mike Nace on bionews-tx.com] Israel-based Kamada Ltd., a drug development company currently developing plasma-derived protein therapeutics that are chiefly targeting orphan diseases, indicated recently in a press release that their lead drug candidate Alpha-1 Antitrypsin (AAT), which is currently in trials for treatment of...

Hearing Loss From Popular Cystic Fibrosis Antibiotic Could Be Treated With Vitamin Supplements

by BEF

CF Community, CF News, Research

March 19, 2019

SOURCE: Bionews-Tx.com Gentamicin belongs to the amino glycoside class of antibiotics and is used to treat mostly gram negative bacterial infections, including severe ones like pyelonephritis, biliary sepsis, endocarditis, and cystic fibrosis among others. It is a bactericidal antibiotic that binds irreversibly to the 30s subunit...

CFF Therapeutics Extends Drug Development Collaboration via CFF.org

by BEF

CF Community, CF News, Research, Therapies

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 BEF

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 BEF

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 BEF

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 BEF

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 BEF

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 BEF

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 BEF

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...

Author: BEF

Kamada Ltd. Launching New CF Clinical Trial this Summer

Hearing Loss From Popular Cystic Fibrosis Antibiotic Could Be Treated With Vitamin Supplements

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

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