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How Astrophysics Could Transform The Treatment Of Cystic Fibrosis And Other Rare Diseases - Latest News


How astrophysics could transform the treatment of cystic fibrosis and other rare diseases

It’s a cruel disease which dramatically shortens life expectancy. One in 25 Europeans carry the cystic fibrosis gene and, in the UK, about 10,400 people currently have the condition. But people are li... read more

‘We’re still waiting’: As cystic fibrosis drugs deliver new hope, not everyone is being swept up by scientific progress

The Cystic Fibrosis Foundation has recognized the splintering of the community and last year dedicated $90 million for research and drug development programs for CF caused by nonsense or other rare .. ... read more

Chemists discover new signatures to identify cystic fibrosis in infants sooner

Credit: JD Howell, McMaster University Scientists at McMaster University have discovered several new biomarkers from a single drop of blood that could allow earlier and more definitive detection of cy... read more

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The challenges of making genomics a reality for patients

Better, safer and targeted treatments for patients. Fast-forward 18 years and more than ever we see the potential of how precision medicine could transform ... or other diseases where there are often ... read more


Eloxx Pharmaceuticals Announces that Two Abstracts for ELX-02 Have Been Accepted for Presentation at the 41st European Cystic Fibrosis Conference

These patients have a high burden of disease, and few, if any, treatment options available,” said Dr. Pedro Huertas, Chief Medical Officer of Eloxx Pharmaceuticals. “We plan to initiate a Phase 2 clin... read more

More cystic fibrosis patients to get Kalydeco in England, but delay criticised

More patients with the life-limiting disease cystic fibrosis are to receive new treatment ... could normalise life expectancy – but only a subset of patients with certain gene mutations have been show... read more

Catabasis Pharmaceuticals: My Long Thesis And Analysis

Along with Edasalonexent, the company is working on a pipeline of product candidates for treatments for other rare diseases. The company's pipeline includes CAT-5571, for cystic fibrosis ... a unique ... read more

Tackling the Cause of Cystic Fibrosis One Mutation at a Time

This little molecule is changing how some cystic fibrosis patients are treated ... The results are promising so far. These treatments for CF are showing how we may have to deal with genetic diseases i... read more

New Miracle Drugs: What Would You Pay?

Levi Collazo, a biology major at Southwestern Oklahoma State University, has cystic fibrosis (CF ... Vice President of Clinical Development for Rare Diseases at Genzyme told me. A pharmacist must prov... read more

How astrophysics could transform the treatment of cystic fibrosis and other rare diseases

It's a cruel disease which dramatically shortens life expectancy. One in 25 Europeans carry the cystic fibrosis gene and, in the UK, about 10,400 people currently have the condition. But people are li... read more

The challenges of making genomics a reality for patients today, not tomorrow

Better, safer and targeted treatments for patients. Fast-forward 18 years and more than ever we see the potential of how precision medicine could transform ... or other diseases where there are often ... read more



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