Cystic Fibrosis Treatment, Therapy and New Medications & Drug

April 24, 2018April 24, 2018
Springs of Various Sorts: Post-transplant Peace and Euphoria
Columns, Victorious - a Column by Brad Dell
Spring 2017 It was a historic rainfall in Silicon Valley, the likes not seen since the days of Noah ... Read more
April 24, 2018April 24, 2018
Eloxx Planning Phase 2 Studies of ELX-02, Potential CF Nonsense Mutation Treatment
cystic fibrosis
Eloxx Pharmaceuticals announced plans to begin Phase 2 studies of ELX-02, its lead candidate to treat cystic fibrosis (CF) and cystinosis, ... Read more
April 23, 2018April 23, 2018
New UK Cystic Fibrosis Innovation Hub to Speed Research in Treatment
cystic fibrosis, News
The new Cystic Fibrosis Innovation Hub at the University of Cambridge in the U.K. will encompass exciting new research about ... Read more
April 20, 2018April 20, 2018
Lung Stem Cells Discovery in Mice May Benefit Cystic Fibrosis Treatment
cystic fibrosis, News
A new population of lung stem cells capable of repairing the airways following a severe injury was identified in ... 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 ... 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 24, 2018April 24, 2018

Springs of Various Sorts: Post-transplant Peace and Euphoria

Columns, Victorious - a Column by Brad Dell

Spring 2017 It was a historic rainfall in Silicon Valley, the likes not seen since the days of Noah and his big boat. Los Gatos Creek spilled over and ... Read more

April 24, 2018April 24, 2018

Eloxx Planning Phase 2 Studies of ELX-02, Potential CF Nonsense Mutation Treatment

cystic fibrosis

Eloxx Pharmaceuticals announced plans to begin Phase 2 studies of ELX-02, its lead candidate to treat cystic fibrosis (CF) and cystinosis, having finished one round of Phase 1 safety and tolerability ... Read more

April 23, 2018April 23, 2018

New UK Cystic Fibrosis Innovation Hub to Speed Research in Treatment

cystic fibrosis, News

The new Cystic Fibrosis Innovation Hub at the University of Cambridge in the U.K. will encompass exciting new research about treatments and approaches for battling the disease, and take a “moon ... Read more

April 20, 2018April 20, 2018

Lung Stem Cells Discovery in Mice May Benefit Cystic Fibrosis Treatment

cystic fibrosis, News

A new population of lung stem cells capable of repairing the airways following a severe injury was identified in two preclinical studies. These cells’ ability to transform and replace ... 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

Springs of Various Sorts: Post-transplant Peace and Euphoria

Eloxx Planning Phase 2 Studies of ELX-02, Potential CF Nonsense Mutation Treatment

New UK Cystic Fibrosis Innovation Hub to Speed Research in Treatment

Lung Stem Cells Discovery in Mice May Benefit Cystic Fibrosis Treatment

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

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