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Disease Duration, Flare-ups Affect Mental Health of Non-CF Bronchiectasis Patients, Study Finds

People with non-cystic fibrosis (CF) bronchiectasis are at an increased risk for anxiety and depression, and this risk is associated with a higher rate of exacerbations (when symptoms suddenly worsen) and shorter disease duration (time since diagnosis), according to a Turkish study. The study, which analyzed self-reported data and disease severity indices, was published in an article, “Clinical impact of depression and anxiety in patients with non-cystic fibrosis bronchiectasis,” in the journal Tuberculosis and Thorax. According to the World Health Organization, the worldwide prevalence of depression and anxiety is about 4%. Psychosocial factors in people with chronic respiratory symptoms ma

VERTEX’S CF BLOCKBUSTER TRIKAFTA EFFECTIVE IN KIDS 6 TO 11 YEARS OF AGE

Vertex Pharmaceuticals announced the completion of its Phase III trial of Trikafta in children ages 6 through 11 years with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Based on the positive results, the company plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter and other regulatory agencies around the world afterward. Trikafta is a triple combination drug, elexacaftor/tezacaftor/ivacaftor and ivacaftor. It is indicated for CF patients ages 12 years and older who have at least one copy of the F508del mutation in the cys

A case for improved carrier screening

Expanded carrier screening addresses limitations in historical ethnic-based screenings, including potential racial and ethnic bias. Introduction Driven by the cost-effective introduction of next-generation sequencing, prenatal screening for fetal genetic disease has dramatically expanded. Although noninvasive prenatal testing (NIPT) for the common aneuploides (trisomy 21, 18 and 13) has had the greatest uptake and gained the most attention, fetal Mendelian disorders actually affect more pregnancies. For example, trisomy 21 occurs in approximately 1 in 750 live births, trisomy 18 in 1 in 5500, and trisomy 13 even less frequently. Alternatively, autosomal recessive and X linked disorders are e

Vertex to Seek Expanded FDA Approval for Trikafta After Positive Child Safety Data

Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is safe for use in children with cystic fibrosis (CF) as young as age 6, data from a global Phase 3 clinical trial show. Based on these results, Vertex Pharmaceuticals, which markets Trikafta, will seek an expansion of the medication’s current U.S. approval, which covers patients ages 12 and older. Vertex is planning to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA). That sNDA is expected to be submitted by the end of the year, with submissions to other regulatory agencies around the world to follow. “Our aim is to extend eligibility to all patients who may benefit from this transforma

Vertex’s Kaftrio approved for use in Europe and made accessible on the NHS

The European Commission and European Medicines Agency have approved the triple combination Kaftrio for use in certain cystic fibrosis patients, triggering the drug to be accessible on the UK’s National Health Service. The European Commission (EC) has approved Vertex Pharmaceuticals’ Kaftrio (ivacaftor/tezacaftor/elexacaftor) for use in combination with ivacaftor to treat people aged 12 plus with certain forms of cystic fibrosis (CF). The decision allows doctors to prescribe the drug when people with the disease are carrying one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This

Age May Affect Outcomes in Lung Transplant Recipients With Cystic Fibrosis

Age may influence outcomes in lung transplant recipients with cystic fibrosis (CF), according to study results published in the Annals of the American Thoracic Society. Lung transplant is the only effective treatment option for end-stage lung disease for patients with CF, and this population has superior posttransplant survival rates compared with other pulmonary conditions. However, according to the study authors, studies have suggested that an age-related disparity exists in patients with CF. Therefore, researchers investigated the effect of age at transplant on posttransplant outcomes in adult patients with CF. A total of 3881 patients with CF underwent lung transplantation between 1992 a

CFF Funding Will Further ContraFect’s Research Into Alternative for Antibiotics

ContraFect will receive funding from the Cystic Fibrosis Foundation to conduct preclinical research about the potential of direct lytic agents, an alternative to conventional antibiotics, to treat lung infections caused by Gram negative antibiotic-resistant bacteria, including Pseudomonas aeruginosa. While antibiotics have had a profound impact on the eradication of otherwise deadly infections, their overuse has fostered the spread of antibiotic-resistant bacteria. Pseudomonas aeruginosa is one of the most critical bacteria affecting cystic fibrosis (CF) patients. In some cases, the bacteria can become resistant to the body’s immune system and to the action of antibiotics. ContraFect direct

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