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November 22, 2019November 22, 2019

French Authorities Approve Reimbursement of Orkambi for CF Patients 2 Years and Older

cystic fibrosis, News

The Comité Économique des Produits de Santé (CEPS) in France has approved national reimbursement of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for patients with cystic fibrosis (CF) who are 2 years of age ... Read more

November 21, 2019November 21, 2019

NIH Grants $2.7M for Research on Anaerobic Bacteria’s Role in CF Flare-ups

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The National Institutes of Health (NIH) has granted $2.7 million to Michigan State University (MSU) scientist Robert Quinn to investigate, in collaboration with Spectrum Health, the role of anaerobic ... Read more

November 20, 2019November 20, 2019

CF-related Diabetes Affects Girls More Often and at Younger Age than Boys, European Study Shows

cystic fibrosis, News

Girls with cystic fibrosis-related diabetes (CFRD) are exposed to risk factors of early death — including chronic infections, poor lung function, and poor nutrition — at a significantly higher proportion and ... Read more

November 19, 2019November 19, 2019

UI Researchers Develop Breakthrough Approach to Gene Therapies for Respiratory Diseases

cystic fibrosis, News

A new, more efficient method for delivering gene therapies to lung tissues — based on tiny engineered proteins, called peptides —is creating therapeutic opportunities for people with diseases like ... Read more

November 18, 2019November 18, 2019

Discovered Bacteria ‘Wars’ May Lead to New Antimicrobial Treatments for CF, Study Suggests

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Pseudomonas aeruginosa, a bacteria that commonly causes serious lung infections in people with cystic fibrosis (CF), reacts to other dangerous bacteria in ways that could be exploited to develop ... Read more

November 15, 2019November 15, 2019

Patients Adhere Well to CFTR Therapies But High Costs Could Undermine Use, Pharmacy Study Says

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People with cystic fibrosis (CF) show a “high adherence” to CFTR modulators, but these newer medications are costly and could undermine compliance to treatment over time, according to a ... Read more

November 14, 2019November 14, 2019

Vertex Reaches Agreement for CF Medications in Northern Ireland and Wales

cystic fibrosis, News

The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) Wales have reached an agreement with Vertex Pharmaceuticals that will allow people with cystic fibrosis ... Read more

November 13, 2019November 13, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

cystic fibrosis, News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

November 12, 2019November 12, 2019

Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis

cystic fibrosis, News

Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who ... Read more

November 11, 2019November 11, 2019

Latinos on 2 Caribbean Islands Carry CFTR Mutations Seen in 1% of CF Patients Elsewhere, Study Says

cystic fibrosis, News

Latinos in the Caribbean with cystic fibrosis (CF) carry rare CFTR gene mutations not often seen in Latin or other CF populations elsewhere, a study led by physician-scientists at ... Read more

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