Emma Mueller, Author at The Boomer Esiason Foundation

March 19, 2019

SOURCE - OFFICIAL PRESS RELEASE Vertex Announces Significant Progress in Its Development Efforts to Treat the Cause of Cystic Fibrosis in the Vast Majority of People with the Disease-Two next-generation correctors to enter clinical development in November; studies of a triple combination of a next-generation corrector...

ORKAMBI™ Is Now FDA Approved!

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should not be used in patients other than those who have two...

CFFT Grants $5 Million to Celtaxsys’ Phase 2 Cystic Fibrosis Study via CF News Today

by Emma Mueller

CF News, Research, Therapies

March 19, 2019

ORIGINAL PRESS RELEASE Celtaxsys has announced that the company’s phase 2 clinical trial to evaluate CTX-4430, its investigational treatment forcystic fibrosis (CF), will be supported by a $5 million grant from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). The CFFT is the non-profit drug discovery and development affiliate...

FDA Panel Recommends Approval of ORKAMBI™ via Vertex

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE --- Please read the original press release here --- SOURCE FDA decision expected by July 5, 2015 PDUFA date Approximately 8,500 people with cystic fibrosis in the U.S. have two copies of the F508del mutation and are ages 12 and older BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)...

Yale scientists use gene editing to correct mutation in cystic fibrosis via Yale News

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. Cystic fibrosis is an inherited, life-threatening disorder that damages the lungs and digestive system. It is most commonly caused...

New antibiotic teixobactin kills drug-resistant superbugs, study says via LA Times

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE: LA TIMES Using soil from a grassy field in Maine and a miniaturized diffusion chamber, scientists have cultivated a microbe that could help tame the spread of antibiotic-resistant superbugs. When tricked into growing in a lab, the microbe makes a compound that kills strains of tuberculosis,...

CFF Announces Unprecedented Acceleration and Expansion of CF Research, Care and Patient Programs

by Emma Mueller

CF Community, CF News, Research

March 19, 2019

SOURCE: CFF.org The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation’s royalty rights to CF treatments developed by Vertex Pharmaceuticals...

Vertex Submits Application for Combination Drug for People with 2 Copies of F508del Mutation

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE - VERTEX OFFICIAL PRESS RELEASE November 5, 2014 Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation -U.S. submission includes request for Priority Review; Accelerated Assessment has...

FDA Recommends Approval of KALYDECO for Use in People with CF R117H Mutation

by Emma Mueller

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE: VERTEX PRESS RELEASE FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration's Pulmonary Allergy Drugs...

Median Age Of Survival For Canadians With CF Surpasses 50 via CF News Today

by Emma Mueller

CF News, Research, Therapies

March 19, 2019

SOURCE In the 1980s, life expectancy for persons with cystic fibrosis (CF) was just 12 years in the US, and around 20 in Canada. However, the median age of survival for Canadian CF patients continues on an upward trend, and is currently estimated to be 50.9...

Author: Emma Mueller

Vertex Announces Significant Progress in Development via Vertex

ORKAMBI™ Is Now FDA Approved!

CFFT Grants $5 Million to Celtaxsys’ Phase 2 Cystic Fibrosis Study via CF News Today

FDA Panel Recommends Approval of ORKAMBI™ via Vertex

Yale scientists use gene editing to correct mutation in cystic fibrosis via Yale News

New antibiotic teixobactin kills drug-resistant superbugs, study says via LA Times

CFF Announces Unprecedented Acceleration and Expansion of CF Research, Care and Patient Programs

Vertex Submits Application for Combination Drug for People with 2 Copies of F508del Mutation

FDA Recommends Approval of KALYDECO for Use in People with CF R117H Mutation

Median Age Of Survival For Canadians With CF Surpasses 50 via CF News Today

Never miss an update. Subscribe to our emails!