2019 was a promising year for cystic fibrosis (CF) patients as a new drug called Trikafta was approved by the FDA and can benefit up to 90% of the patient population. It works with those patients that share the most common mutation (delta-F508) to the CFTR gene. Local...
Cystic Fibrosis
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Life or Death for Young Lady with Cystic Fibrosis—Trikafta Not Approved in UK & Europe and Time’s Running Out
28 year-old Nicole Adams from Belfast is literally being suffocated by her own body due to her case of cystic fibrosis. A wonder drug called Trikafta could potentially save her. The problem: although approved in America, it hasn’t been approved in Europe or the UK;...
University of Kansas Health System Contributes to the Successful Phase III Cystic Fibrosis Treatment now FDA Approved
The University of Kansas Health System is one 115 global sites participating in a worldwide study for a new treatment considered by some as a “game-changer.” Focusing on a new therapy for cystic fibrosis, the new treatment was invented in part by the Cystic Fibrosis...
FDA Approves New Breakthrough Therapy for Cystic Fibrosis
The U.S. Food and Drug Administration recently approved Vertex Pharmaceutical’s Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for...
$15M into University of Queensland Cystic Fibrosis Research Collaborative in Australia
Queensland’s first cystic fibrosis research program will be established to help improve outcomes for patients living with the life-threatening genetic disorder in Australia. The Queensland Cystic Fibrosis Research Program receives $15 million form the University of...
Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis, as Reported By the Study’s Author
Pierre-Régis Burgel MD, PhD, Professor of Respiratory Medicine at Paris Descartes University and Coordinator of French National Reference Center for Cystic Fibrosis at Cochin Hospital, interviewed with MedicalResearch.com regarding the study of lumacaftor-ivacaftor...
Margaret Rosenfeld, MD, MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine
Dr. Rosenfeld was a lead investigator in the Phase 3 ARRIVAL study, sponsored by Vertex, which found Ivacaftor (KALYDECO®) is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. Dr. Rosenfeld noted...
Telethon Kids Institute Spin-off Receives Sizable Investment to Test Cystic Fibrosis Treatment
Respirion Pharmaceuticals, a spin-off of the Telethon Kids Institute, will receive an investment of over $20 million from the Australian government’s Medical Research Commercialization Fund (MRCF) as well as the US Cystic Fibrosis Foundation. The spin-off company has...
Children’s Hospital Chicago Researchers Work Towards Eventual Precision Medicine for Cystic Fibrosis
Researchers from the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago have identified distinct gene expression patterns that could lead to patient-tailored treatments in the future. The Chicago research group...
Vertex Pharma Refuses to Supply 3-Year Old with Cystic Fibrosis Drug
The New York Post reports that Scrooge should move over for New England drug maker Vertex. As reported, a Brooklyn family now may spend Christmas in the hospital with their 3-year old daughter hooked up to a feeding tube because Vertex won’t give her a three-week...
Children’s Hospital Chicago Study Paves Way for Precision Medicine for CF
As reported in EurekAlert! researchers at Stanley Manne Children’s Research Institute at Ann Rogert H. Lurie Children’s Hospital of Chicago and colleagues used a blood test and microarray technology to identify distinct molecular signatures in children with cystic...
Children’s Colorado Investigators Identify Risk Factors of Advanced Liver Disease in Cystic Fibrosis
Children’s Hospital Colorado reported in EurekAlert! that a research team has helped to identify factors tied to greater risk of developing advanced liver disease in cystic fibrosis patients. Michael Narkwewicz, MD recently shared the results of a Prospective Study of...
USC Center for Cystic Fibrosis Receives $1 Million from Anton Yelchin Foundation
The USC Center for Cystic Fibrosis – Adult Care at Keck Hospital of USC has received a generous $1 million gift from the Anton Yelchin Foundation. Anton Yelchin had cystic fibrosis and the USC Center for Cystic Fibrosis – Adult Care provided 10 years of care to help...
Price Watch: Univ.of Alabama & Denver NJH Drives Cystic Fibrosis Research Trials
Ivacaftor–$300,000 per year
Vertex and Genomics Team Up in Machine Learning-Driven Quest for Genetics -Based Precision Medicines
Cystic Fibrosis News Today reports that Vertex Pharmaceuticals and Genomics announced a three-year collaboration to better understand genetic variations and their effect on clinical outcomes of diseases with unmet therapeutic needs, including cystic fibrosis. The...
Another Australian Breakthrough: Stem Cell Research for Cystic Fibrosis Moves Forward
University of Adelaide reports that the fight against cystic fibrosis has taken a major step forward, with new research showing that cells causing the debilitating genetic disorder could be successfully replaced with healthy ones. The fight against cystic fibrosis...
Children’s Hospital of Colorado Key Investigative Site in Vertex’s Potential Triple Combo CF Therapy
As reported in Cystic Fibrosis News Today, Vertex is running a phase 3 trial with up to 82 investigative sites with Children’s Hospital Colorado’s Mike Morris Cystic Fibrosis Research and Care Center playing a key role in supporting the study. As reported by Alice...
Cystic Fibrosis Breakthrough Treatment Study
Kristi Rosa of RareDisease Report notes that over 70,000 are effected by cystic fibrosis and "although advances have been made in the fight against the progressive genetic disease which has allowed these patients to live longer lives, additional treatment options are...
Potential Breakthrough Cystic Fibrosis Therapy in Phase 3 Trial
Cystic fibrosis, a genetic disorder impacting not only the lungs but also the pancreas, liver,kidneys and intestines.1 in 3000 may inherit cystic fibrosis in an autosomal recessive manner--originating from presence of mutations in both copies of the gene for the...