Cystic Fibrosis Treatment, Therapy and New Medications & Drug

April 19, 2018April 19, 2018
CFF’s Therapeutics Development Arm Endorses Trial of PTI-428, Symdeko Combo
cystic fibrosis, News
The Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation has endorsed Proteostasis Therapeutics‘ trial assessing PTI-428 in combination with Symdeko ... Read more
April 18, 2018April 18, 2018
Enterprise Therapeutics Raises $41M to Advance Mucus-targeting Therapies for CF
cystic fibrosis, News
Enterprise Therapeutics recently closed a £29 million ($41 million) Series B financing round to help advance into clinical development ... Read more
April 17, 2018April 17, 2018
When Invisible Illness Becomes Visible
Columns, Victorious - a Column by Brad Dell
I breathed 23 years of invisible illness before retching tan-green Silly Putty into city trash bins while leashed to ... Read more
April 17, 2018April 17, 2018
Translate Bio Readies 1st Clinical Trial of MRT5005, Potential Treatment for All CF Patients
cystic fibrosis, News
The U.S. Food and Drug Administration has given the go-ahead for MRT5005, Translate Bio’s lead candidate to treat the ... Read more
April 16, 2018April 16, 2018
Autogenic Drainage Physiotherapy Can Improve Lung Respiratory Mechanisms, Study Shows
cystic fibrosis, News
Autogenic drainage physiotherapy can effectively improve the underlying lung mechanical impairment of cystic fibrosis (CF), a study shows. Respiratory ... Read more
April 13, 2018April 13, 2018
Orkambi’s Components Trigger Liver Enzyme That Decreases Its Ability to Fight CF, Study Finds
cystic fibrosis, News
The two components of Orkambi act separately to trigger a liver enzyme that decreases the therapy’s ability to fight cystic fibrosis, ... Read more
April 12, 2018April 12, 2018
NIH Agency Pioneers Collaborative Research into Rare Diseases
cystic fibrosis
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed ... Read more
April 12, 2018April 12, 2018
Abnormal Glucose Levels Linked to Poorer Lung Function in Cystic Fibrosis Youth, Study Shows
cystic fibrosis, News
Abnormalities in continuous glucose monitoring (CGM) measures correlate with a decline in lung function in children and adolescents with ... Read more
April 11, 2018April 11, 2018
Bacteria Can Pass Memory of Biofilm Formation to Descendants, Study Shows
cystic fibrosis, News
Bacteria can transmit memory across generations so their descendants will know how to attach themselves to surfaces and form ... Read more
April 10, 2018April 10, 2018
What Is Victory? Sharing Our Reality.
Columns, Victorious - a Column by Brad Dell
What is victory for us? I know we crave a cure above all, but what is attainable in the ... Read more
April 10, 2018April 10, 2018
CF Foundation Opposes Lastest Version of Right to Try Act as Still Posing Risks
cystic fibrosis, News
The Cystic Fibrosis Foundation (CFF) has joined 70 other organizations in signing a letter opposing the latest version of ... Read more
April 6, 2018April 6, 2018
2 Urine Biomarkers Can Help Detect Aminoglycosides-induced Kidney Damage in CF Patients, Study Suggests
cystic fibrosis
Quantification of two specific molecules in urine can help in the early detection of kidney damage triggered by treatment ... Read more

April 19, 2018April 19, 2018

CFF’s Therapeutics Development Arm Endorses Trial of PTI-428, Symdeko Combo

cystic fibrosis, News

The Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation has endorsed Proteostasis Therapeutics‘ trial assessing PTI-428 in combination with Symdeko (tezacaftor/ivacaftor) as a treatment for cystic fibrosis (CF). The Phase 2 trial ... Read more

April 18, 2018April 18, 2018

Enterprise Therapeutics Raises $41M to Advance Mucus-targeting Therapies for CF

cystic fibrosis, News

Enterprise Therapeutics recently closed a £29 million ($41 million) Series B financing round to help advance into clinical development the company’s pipeline of potential mucus-targeting therapies for cystic fibrosis ... Read more

April 17, 2018April 17, 2018

When Invisible Illness Becomes Visible

Columns, Victorious - a Column by Brad Dell

I breathed 23 years of invisible illness before retching tan-green Silly Putty into city trash bins while leashed to a bulky, wheeled, metallic canister of oxygen. Twenty-three years of ... Read more

April 17, 2018April 17, 2018

Translate Bio Readies 1st Clinical Trial of MRT5005, Potential Treatment for All CF Patients

cystic fibrosis, News

The U.S. Food and Drug Administration has given the go-ahead for MRT5005, Translate Bio’s lead candidate to treat the underlying cause of cystic fibrosis (CF), to be tested in ... Read more

April 16, 2018April 16, 2018

Autogenic Drainage Physiotherapy Can Improve Lung Respiratory Mechanisms, Study Shows

cystic fibrosis, News

Autogenic drainage physiotherapy can effectively improve the underlying lung mechanical impairment of cystic fibrosis (CF), a study shows. Respiratory physiotherapy is an important part of the treatment of patients ... Read more

April 13, 2018April 13, 2018

Orkambi’s Components Trigger Liver Enzyme That Decreases Its Ability to Fight CF, Study Finds

cystic fibrosis, News

The two components of Orkambi act separately to trigger a liver enzyme that decreases the therapy’s ability to fight cystic fibrosis, a study reported. Vertex Pharmaceuticals created Orkambi by combining ivacaftor and lumacaftor ... Read more

April 12, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

cystic fibrosis

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 12, 2018April 12, 2018

Abnormal Glucose Levels Linked to Poorer Lung Function in Cystic Fibrosis Youth, Study Shows

cystic fibrosis, News

Abnormalities in continuous glucose monitoring (CGM) measures correlate with a decline in lung function in children and adolescents with cystic fibrosis (CF), a study suggests. The study, “Continuous glucose ... Read more

April 11, 2018April 11, 2018

Bacteria Can Pass Memory of Biofilm Formation to Descendants, Study Shows

cystic fibrosis, News

Bacteria can transmit memory across generations so their descendants will know how to attach themselves to surfaces and form communities called biofilms, a study reports. Researchers’ findings shed light ... Read more

April 10, 2018April 10, 2018

What Is Victory? Sharing Our Reality.

Columns, Victorious - a Column by Brad Dell

What is victory for us? I know we crave a cure above all, but what is attainable in the meantime? When I began this column, titled “Victorious,” I viewed ... Read more

April 10, 2018April 10, 2018

CF Foundation Opposes Lastest Version of Right to Try Act as Still Posing Risks

cystic fibrosis, News

The Cystic Fibrosis Foundation (CFF) has joined 70 other organizations in signing a letter opposing the latest version of the Right to Try Act, which the House passed recently. It ... Read more

April 6, 2018April 6, 2018

2 Urine Biomarkers Can Help Detect Aminoglycosides-induced Kidney Damage in CF Patients, Study Suggests

cystic fibrosis

Quantification of two specific molecules in urine can help in the early detection of kidney damage triggered by treatment with aminoglycosides in patients with cystic fibrosis (CF), a study ... Read more

CFF’s Therapeutics Development Arm Endorses Trial of PTI-428, Symdeko Combo

Enterprise Therapeutics Raises $41M to Advance Mucus-targeting Therapies for CF

When Invisible Illness Becomes Visible

Translate Bio Readies 1st Clinical Trial of MRT5005, Potential Treatment for All CF Patients

Autogenic Drainage Physiotherapy Can Improve Lung Respiratory Mechanisms, Study Shows

Orkambi’s Components Trigger Liver Enzyme That Decreases Its Ability to Fight CF, Study Finds

NIH Agency Pioneers Collaborative Research into Rare Diseases

Abnormal Glucose Levels Linked to Poorer Lung Function in Cystic Fibrosis Youth, Study Shows

Bacteria Can Pass Memory of Biofilm Formation to Descendants, Study Shows

What Is Victory? Sharing Our Reality.

CF Foundation Opposes Lastest Version of Right to Try Act as Still Posing Risks

2 Urine Biomarkers Can Help Detect Aminoglycosides-induced Kidney Damage in CF Patients, Study Suggests

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