Emma Mueller, Author at The Boomer Esiason Foundation

March 19, 2019

Source: Cystic Fibrosis Foundation An expanded access program for a potential new CF drug, Kalydeco™ (VX-770), is now available at participating clinical sites throughout the country for people with the G551D mutation who have highly limited lung function and may benefit from treatment. Vertex Pharmaceuticals, Inc., the...

Mannitol Shown to Improve Lung Function When Added to Standard CF Therapy

by Emma Mueller

CF News, Therapies

March 19, 2019

Source: American Thoracic Society press release Adding inhaled dry powder mannitol to standard therapy for cystic fibrosis produced sustained improvement in lung function for up to 52 weeks, according to a new study. Along with the treatment's efficacy and good safety profile, the convenience and ease...

FDA Grants Priority Review for KALYDECO™ from Vertex Pharmaceuticals

by Emma Mueller

CF News, Therapies

March 19, 2019

Source: Vertex Pharmaceuticals press release Vertex Pharmaceuticals Incorporated announced today that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECO™ (ivacaftor) and granted the company's request for six-month priority review. KALYDECO targets the defective protein that causes cystic fibrosis (CF)...

How CF-Related Diabetes Differs from Types 1 and 2

by Emma Mueller

CF News, Therapies

March 19, 2019

Source: Diabetic Live In addition to Type 1 and Type 2 diabetes, there is another variant of the disease: cystic fibrosis-related diabetes. It differs in some important ways from Type 1 and Type 2 diabetes and it requires different treatment methods. Patients with cystic fibrosis experience decreased...

New England Journal of Medicine Features Study of Kalydeco (VX-770)

by Emma Mueller

CF News, Therapies

March 19, 2019

Source: Cystic Fibrosis Foundation The New England Journal of Medicine, the world’s most widely read and influential medical periodical, features a study this week about a Phase 3 clinical trial of Kalydeco™ (VX-770), a potential CF therapy undergoing review for approval by the U.S. Food and...

Vertex Applies to FDA for Priority Review, Approval of VX-770

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Inc. today announced the submission of a new drug application to the U.S. Food and Drug Administration for KALYDECO™ (VX-770, ivacaftor), a medicine in development that targets the defective protein that causes cystic fibrosis. KALYDECO (kuh-LYE-deh-koh) was studied among people with CF ages six...

FDA Requests Additional Study of Arikace®, Insmed Says

by Emma Mueller

CF News, Therapies

March 19, 2019

Insmed Inc., a biopharmaceutical company, today announced that it has been notified by the U.S. Food and Drug Administration that it is continuing the clinical hold previously placed on Insmed's phase 3 clinical trials for ARIKACE® (liposomal amikacin for inhalation) in cystic fibrosis patients with...

Pulmatrix Invests in Clinical Programs in Cystic Fibrosis and COPD

by Emma Mueller

CF News, Therapies

March 19, 2019

Pulmatrix, a clinical stage biotechnology company discovering and developing a new class of therapies for the prevention, treatment and control of respiratory diseases, today announced that its clinical development plans will focus on the company’s lead clinical candidate in chronic obstructive pulmonary disease (COPD) and cystic...

Insmed Announces Clinical Hold on ARIKACE® Phase 3 Clinical Trials

by Emma Mueller

CF News, Therapies

March 19, 2019

Insmed Inc., a biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has notified the company that the agency has placed a clinical hold on Insmed's phase 3 clinical trials for ARIKACE® (liposomal amikacin for inhalation) in cystic fibrosis (CF) patients with Pseudomonas...

Vertex Plans to Submit New Drug Application for VX-770 to FDA in October

by Emma Mueller

CF News, Therapies

March 19, 2019

Vertex Pharmaceuticals Inc. today announced it plans to submit to the U.S. Food and Drug Administration in October a new drug application for VX-770, a promising CF therapy. Following is information on the announcement from Vertex and the Cystic Fibrosis Foundation. FROM THE CYSTIC FIBROSIS FOUNDATION...

Author: Emma Mueller

Kalydeco (VX-770) Available to People with Critical Medical Need

Mannitol Shown to Improve Lung Function When Added to Standard CF Therapy

FDA Grants Priority Review for KALYDECO™ from Vertex Pharmaceuticals

How CF-Related Diabetes Differs from Types 1 and 2

New England Journal of Medicine Features Study of Kalydeco (VX-770)

Vertex Applies to FDA for Priority Review, Approval of VX-770

FDA Requests Additional Study of Arikace®, Insmed Says

Pulmatrix Invests in Clinical Programs in Cystic Fibrosis and COPD

Insmed Announces Clinical Hold on ARIKACE® Phase 3 Clinical Trials

Vertex Plans to Submit New Drug Application for VX-770 to FDA in October

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