Cystic Fibrosis Care Still Below Pre-Pandemic Levels
New data shows that patient visits and lung function tests for cystic fibrosis have not returned to 2019 levels despite new treatments and telehealth options.
Tag: CFTR
All
Latest
Cystic Fibrosis Care Still Below Pre-Pandemic Levels
New data shows that patient visits and lung function tests for cystic fibrosis have not returned to 2019 levels despite new treatments and telehealth options.
Read More
CFTR Modulator Use Linked to Lower Anxiety in Children and Parents
New research suggests cystic fibrosis transmembrane conductance regulator (CFTR) modulators may provide mental health benefits for pediatric patients and their caregivers early in treatment.
Read More
Nanoparticle-based Gene Editing for Cystic Fibrosis
Researchers developed lipid nanoparticles capable of inserting a full healthy CFTR gene into lab-grown human airway cells with severe cystic fibrosis.
Read More
Reevaluating Salt Intake for Cystic Fibrosis Patients
Researchers assessed salt intake in cystic fibrosis patients undergoing treatment with CFTR modulators, which improve salt conservation and other physiological markers.
Read More
Cystic Fibrosis and Abnormal Lung Function
Changes in lung function are of critical importance to patients with cystic fibrosis (CF), as lung function is a key indicator of disease progression and treatment effectiveness.
Read More
FDA Approves Alyftrek CFTR Modulator for Cystic Fibrosis
The FDA approved Alyftrek, a once-daily triple combo CFTR modulator, for certain cystic fibrosis patients aged 6 and older.
Read More
CF Foundation Invests $15M in ReCode to Develop Gene Editing Therapy
The Cystic Fibrosis Foundation is funding the development of a gene editing therapy that is designed to use ReCode’s tissue-specific delivery vehicle to transport gene editing cargo to the lung cells in people with cystic fibrosis.
Read More
Phase 1 Study of Inhaled Peptide Therapy for Cystic Fibrosis Begins
The study will evaluate the safety and tolerability of an inhaled peptide therapy designed to enhance CFTR function, act as a bronchodilator, and reduce inflammation.
Read More
FDA Greenlights Phase 2 Study of Inhaled mRNA Treatment for Cystic Fibrosis
The phase 2 study will evaluate the safety and efficacy of ARCT-032, an inhaled mRNA therapy for cystic fibrosis patients, which previously received Orphan Drug Designation.
Read More
First Patients Dosed in Two Phase 1 Trials for Novel Cystic Fibrosis Treatments
The phase 1 trials are evaluating two NBD1 stabilizers, SION-719 and SION-451, which are designed to improve CFTR protein function in cystic fibrosis patients.
Read More
Vertex’s Next-in-class Triple Combo Therapy for Cystic Fibrosis Gets FDA Priority Review
The drug candidate “raises the high bar set by Trikafta,” according to Vertex Pharma’s executive vice president and chief regulatory and quality officer.
Read More
Scientists Correct Cystic Fibrosis Genes in Mice for Almost Two Years
The scientists developed nanoparticles that successfully edited the disease-causing gene in the lungs of a mouse model of cystic fibrosis.
Read More
FDA Fast Tracks Investigational Cystic Fibrosis Drug
SPL84, an antisense oligonucleotide treatment for cystic fibrosis patients with a specific mutation, gains FDA Fast Track designation.
Read More
Ivacaftor Safe in CF Infants Aged 1 to 4 Months
A phase 3 clinical trial, ivacaftor (Kalydeco, Vertex Pharma) was generally safe to administer to infants aged 1-month to <4-months old, according to research published in Journal of Cystic Fibrosis.
Read More
Zinc Manipulation Could Restore Immune Defense in Cystic Fibrosis
University of Queensland researchers have discovered a fault in the bacteria-killing function of immune cells in people with cystic fibrosis and a potential way to get around it.
Read More
Cystic Fibrosis Foundation Funds Prime Medicine’s Gene Editing Research
The Cystic Fibrosis Foundation will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis.
Read MoreHealth Canada OKs Trikafta for Younger Children With Cystic Fibrosis
Health Canada granted market authorization for the expanded use of Trikafta to include children with cystic fibrosis ages 2-5 years who have at least one copy of the F508del mutation in the CFTR gene.
Read More
Disease-causing Stem Cells Dominate Lungs of Advanced CF Patients
Five lung stem cell variants dominate the lungs of patients with advanced cystic fibrosis (CF), and these variants drive key aspects of CF pathology including inflammation, fibrosis, and mucin secretion, according to new research.
Read More
Cystic Fibrosis mRNA Therapeutic Candidate Gets Funding Boost
The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test ARCT-032, an inhaled messenger RNA therapeutic candidate that could treat the underlying cause of cystic fibrosis in all people living with the disease.
Read More
CFTR Modulators Redefining Cystic Fibrosis Care in the Youngest Patients
Clinicians share best practices for implementing CFTR modulators in some of their youngest patients, while research explores their potential to lessen the burden of care.
Read More
RT 2023 August-September: The Digital Edition
In RT’s August-September 2023 issue, we provide an update on the most commonly prescribed pharmacological therapies for respiratory disorders like COPD and asthma; use of CFTR modulators for cystic fibrosis; And ICU/ED aerosol delivery protocols for asthma.
Read MoreHow a Cystic Fibrosis Urine Test Can Help Monitor Patients
A newly-developed cystic fibrosis urine test can make it much easier for doctors to diagnose how badly a patient is affected by the hereditary diseases.
Read More
Predicting Pediatric Cystic Fibrosis Events
The combined use of patient-reported symptom data and objective lung function measures may help manage acute respiratory events in pediatric CF patients.
Read More
Study: Restoring the Airways’ Seal in CF
Using a model reproducing a respiratory epithelium, teams from the University of Geneva have discovered that a film of liquid is sufficient to restore the airways’ seal and lower the risk of bacterial infection.
Read More