Emma Mueller, Author at The Boomer Esiason Foundation

March 19, 2019

Vertex Pharmaceuticals Inc. announced a plan to provide VX-770, a CF medicine in development, to people with the G551D mutation who are in critical medical need and may benefit from treatment prior to potential approval of the drug from the U.S. Food and Drug Administration...

VX-770 Shows Positive and Lasting Results Throughout 48-Week Phase 3 Study

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Inc. today announced final results of a Phase 3 clinical trial of VX-770, a CF medicine in development that targets the underlying cause of the disease. Participants who took the drug had rapid and significant improvements in lung function and other key measures...

Phase 2 Study of VX-770 and VX-809 Show Promising Results in Patients with Most Common Mutation

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Incorporated and the Cystic Fibrosis Foundation today announced promising results from an ongoing Phase 2 study evaluating combinations of VX-770 and VX-809, potential medicines designed to treat the defective protein that causes cystic fibrosis. The study enrolled 62 people with two copies of the...

Aradigm Gets FDA Orphan Drug Designation for Ciprofloxacin for Inhalation in Bronchiectasis

by Emma Mueller

CF News, Therapies

March 19, 2019

Aradigm Corporation today announced it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for ciprofloxacin for inhalation for the management of bronchiectasis (BE). Previously, Aradigm received orphan drug designations from FDA for liposomal ciprofloxacin for inhalation for the management of bronchiectasis...

Drug Shortages Force Hospitals to Hunt for Substitutes

by Emma Mueller

CF Community, CF News, Therapies

March 19, 2019

A growing shortage of medications for a host of illnesses — from cancer to cystic fibrosis to cardiac arrest — has hospitals scrambling for substitutes to avoid patient harm, and sometimes even delaying treatment. "It's just a matter of time now before we call for a...

New CF Wind Sprint Demonstrates How to Use the PowerLung

by Emma Mueller

Boomer Esiason Foundation, CF News, Therapies

March 19, 2019

As part of its CF Awareness Month focus on exercise, the Boomer Esiason Foundation has released Episode 17 in the Cystic Fibrosis Wind Sprint series: Using the PowerLung. In this video, BEF Volunteer Jerry Cahill demonstrates how to use the PowerLung, an easy-to-use device that can help increase lung...

WBUR Special Report on VX-770

by Emma Mueller

CF News, Therapies

March 19, 2019

by Carey Goldberg, WBUR's "Common Health" [Note: This is a "good news" story. It describes the scientific road to the first drug that successfully attacks the underlying defect in cystic fibrosis, bringing dramatic improvements. But the drug appears to work for only 4% of cystic fibrosis...

FDA Rejects Liprotamase, Calls for More Studies

by Emma Mueller

CF News, Therapies

March 19, 2019

In a widely expected move, the U.S. Food and Drug Administration has rejected a drug to treat cystic fibrosis made by Alnara Pharmaceuticals, which is owned by Indianapolis-based Eli Lilly and Co. The FDA has ordered additional clinical trials to further study the drug candidate,...

CFF Expands Vertex Partnership

by Emma Mueller

CF News, Therapies

March 19, 2019

Today the Cystic Fibrosis Foundation announced the expansion of its collaboration with Vertex Pharmaceuticals for the discovery and development of additional drugs aimed at treating the underlying cause of cystic fibrosis. The new program will support development of a potential new drug called VX-661, designed to...

Vertex Press Release on VX-770 Phase 3 Clinical Trial

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Incorporated today announced positive results from a 24-week analysis of the ongoing Phase 3 ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate VX-770 among children ages...

Author: Emma Mueller

Vertex to Expand Access to VX-770 for Patients with Critical Medical Need

VX-770 Shows Positive and Lasting Results Throughout 48-Week Phase 3 Study

Phase 2 Study of VX-770 and VX-809 Show Promising Results in Patients with Most Common Mutation

Aradigm Gets FDA Orphan Drug Designation for Ciprofloxacin for Inhalation in Bronchiectasis

Drug Shortages Force Hospitals to Hunt for Substitutes

New CF Wind Sprint Demonstrates How to Use the PowerLung

WBUR Special Report on VX-770

FDA Rejects Liprotamase, Calls for More Studies

CFF Expands Vertex Partnership

Vertex Press Release on VX-770 Phase 3 Clinical Trial

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