Posts by: Ana Pena

cystic fibrosis, News

An inhaled form of messenger RNA (mRNA) with the potential to safely deliver therapeutics to the lungs of cystic fibrosis (CF) patients has been discovered by researchers at the ... Read more

December 13, 2018December 13, 2018

Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests

cystic fibrosis, News

The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically measures the sweating dependent on CFTR, called C-sweat rate, than using ... Read more

November 12, 2018November 12, 2018

Liver Disease Doesn’t Worsen Lung Function Decline in CF Patients, Study Reports

cystic fibrosis, News

Cystic fibrosis (CF) patients with liver disease do not experience greater lung function decline compared with CF patients without liver presentation, a study reports. The study, “Analysis of a large ... Read more

November 8, 2018November 8, 2018

Worse CF Lung Outcomes Linked with Mutations Causing Iron Overload Disorder, Study Finds

cystic fibrosis, News

Patients with cystic fibrosis (CF) who carry certain mutations in the HFE gene, which are also behind an iron overload disorder, have poorer lung function that declines faster and ... Read more

November 2, 2018November 2, 2018

Symkevi Approved in EU for CF Patients with Two F508del Mutations, or One Plus a Minimal Function Mutation

cystic fibrosis, News

Symkevi (tezacaftor/ivacaftor combo), by Vertex Pharmaceuticals, has been approved in the European Union for patients with cystic fibrosis (CF) ages 12 or older who carry F508del mutations. Importantly, the European Commission’s decision to grant marketing ... Read more

October 31, 2018October 31, 2018

Santhera Starting Phase 1b/2a Trial Testing Potential CF therapy POL6014

cystic fibrosis, News

A Phase 1b/2a, dose-escalating clinical trial evaluating the effects of POL6014 in patients with cystic fibrosis (CF) was recently launched, according to Santhera Pharmaceuticals, developer of the investigational therapy. The ... Read more

October 30, 2018October 30, 2018

VX-445 Triple Combo Improves Lung Function in CF Patients with F508del Mutations, Phase 2 Trial Shows

cystic fibrosis, News

Adding the investigational therapy VX-445 to tezacaftor (VX-661) and Kalydeco (ivacaftor) led to a roughly 10 percent improvement in the lung function of cystic fibrosis (CF) patients with F508del mutations in the CFTR gene ... Read more

October 29, 2018October 29, 2018

#NACFC2018 — Celtaxsys’ Acebilustat Delays Onset, Reduces Rate of Pulmonary Exacerbations in CF, Phase 2 Study Shows

cystic fibrosis, News

Once-daily acebilustat, an investigational oral therapy for cystic fibrosis (CF) under development by Celtaxsys, reduces the frequency and delays the onset of pulmonary exacerbations in CF patients, results from ... Read more

October 23, 2018October 23, 2018

Santhera’s Potential CF Inhalation Therapy Favored for Orphan Drug Status in EU

cystic fibrosis, News

Santhera Pharmaceuticals’ investigative inhalation therapy for cystic fibrosis (CF), POL6014, has been favored for orphan drug designation within the European Union. A recommendation supporting the designation, which offers financial and regulatory incentives ... Read more

October 17, 2018October 17, 2018

Orkambi May Be More Effective in CF Patients with Inflamed Airways, Study Suggests

cystic fibrosis, News

Two compounds that make up approved cystic fibrosis (CF) therapy Orkambi — lumacaftor and ivacaftor — may work more effectively in patients whose airways are inflamed, according to new research. By ... Read more

Posts by: Ana Pena

Ana Pena

New Inhaled Carrier May Work for CF mRNA Therapy, Mouse Study Finds

Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests

Liver Disease Doesn’t Worsen Lung Function Decline in CF Patients, Study Reports

Worse CF Lung Outcomes Linked with Mutations Causing Iron Overload Disorder, Study Finds

Symkevi Approved in EU for CF Patients with Two F508del Mutations, or One Plus a Minimal Function Mutation

Santhera Starting Phase 1b/2a Trial Testing Potential CF therapy POL6014

VX-445 Triple Combo Improves Lung Function in CF Patients with F508del Mutations, Phase 2 Trial Shows

#NACFC2018 — Celtaxsys’ Acebilustat Delays Onset, Reduces Rate of Pulmonary Exacerbations in CF, Phase 2 Study Shows

Santhera’s Potential CF Inhalation Therapy Favored for Orphan Drug Status in EU

Orkambi May Be More Effective in CF Patients with Inflamed Airways, Study Suggests

Pin It on Pinterest