MECFA Blog

News & Articles

Hyperbaric Oxygen Treatment May Make Antiobiotics More Effective Against P. aeruginosa Infections, S

Exposing cystic fibrosis (CF) patients to high levels of oxygen through hyperbaric oxygen treatment may make Pseudomonas aeruginosa bacterial aggregates more sensitive to antibiotics like tobramycin and easier to eradicate from the lungs, a study in patient-derived bacterial cultures reports. The study, “Hyperbaric oxygen treatment increases killing of aggregating Pseudomonas aeruginosa isolates from cystic fibrosis patients,” was published in the Journal of Cystic Fibrosis. P. aeruginosa is the primary bacterium causing lung infections in CF patients. Tobramycin is an antibiotic often used to treat P. aeruginosa; however, chronic infections by this bacteria — which aggregates in the lungs a

New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows

A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases, including cystic fibrosis (CF), a study reports. This newly developed approach uses man-made RNA molecules, called anticodon engineered transfer RNAs or ACE-tRNA, that prevent the detection of abnormal stop signals in a gene’s sequences. This results in the production of normal-length, functional proteins instead of the shorter protein versions that are associated with several diseases. The study describing the findings, “Engineered transfer RNAs for suppression of premature termination codons,” was published in the scientific journal Nature Communications. Mu

Paying more attention to antibiotic dosing could improve clinical outcomes for CF patients

Download PDF Copy Children and young adults with cystic fibrosis (CF) whose lung infections were treated with suboptimal doses of antibiotics had fewer changes in lung microbial diversity during the IV treatment, and their microbial diversity levels were higher 30 days later, a multi-institutional study that includes Children's researchers shows. By contrast, patients who were treated with therapeutic doses had greater decreases in lung microbial diversity and significantly lower diversity levels when the antibiotic treatment ended as well as 30 days later. "With the subtherapeutic treatment group, this could represent a 'basement effect' where it is harder to decrease diversity when it is a

Savara Provides Update on IMPALA and AVAIL Pivotal Clinical Studies

AUSTIN, Texas, Feb. 06, 2019 (GLOBE NEWSWIRE) -- Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, today provided an update on its two pivotal Phase 3 clinical studies, IMPALA and AVAIL. IMPALA IMPALA is a pivotal Phase 3 clinical study evaluating Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). It is the largest clinical study ever conducted in aPAP, with a total of 139 people across 34 study sites. Savara remains on track to report top line results from the ongoing study at the end of the second quarter 2019. Robust results from IMPALA would fa

Medical Marijuana ‘Can Help Everyone,’ Says Director at Maryland Cannabis Facility

Warning the reporter accompanying him not to take any pictures, veteran horticulturalist Michael Castleman punches an electronic code and unlocks the door to Room 209, nicknamed the “Mother Room.” Photography is indeed forbidden inside this living vault, which contains 20 phenotypes of cannabis plants thriving under the glare of 25 ceramic metal halide lamps for 18 hours a day. The plants, arranged in groups of four and narrowed down from an original 1,000 seeds, bear colorful names like Oro Blanco, Bubblegum Diesel, and Sunshine Daydream. Marijuana plants of the Oro Blanco variety dry in Room 212 of the Kind Therapeutics cannabis cultivation facility in Hagerstown, Maryland. (Photos by Larr

Why you should consider couple genetic screening before pregnancy or marriage

Many genetic disorders happen when someone has two bad (faulty) copies of a gene, one from each parent. In such cases, if one has only one faulty copy, they won’t have symptoms of the genetic condition, but are a “carrier” for it. The baby will be born with the disorder only if both parents are carriers and both pass the bad gene to him. If both parents are carriers for a certain genetic disorder, the chance that they will both pass over the faulty genes to their baby will be 25 per cent. To test if the couple is a carrier of a genetic disease, the doctor usually takes a small sample of their saliva or blood during a checkup before they plan to get pregnant or before marriage, if they plan t

Link Found Between P. aeruginosa Infection and Disease Severity in CF Patients, Study Reports

Patients with cystic fibrosis (CF) whose lungs have never been colonized by the Pseudomonas aeruginosa bacterium have a milder form of the disease, a retrospective study based on registry data suggests. The study, “Clinical and microbiological characteristics of cystic fibrosis adults never colonized by Pseudomonas aeruginosa: Analysis of the French CF registry,” was published in the journal Plos One. P. aeruginosa infections in the airways can cause inflammation and subsequent pulmonary exacerbations that impair lung function over time. Previous reports from national CF registries in the U.S., Canada, France, Germany, Ireland, and the U.K. all suggest that P. aeruginosa prevalence in CF pa

Abstract Submission Deadline is Extended!

The deadline to submit an abstract to the Middle East Cystic Fibrosis Conference has been extended to March 1st, 2019 at 11:59 PM in Istanbul time. https://www.mecfa.org/call-for-abstracts2019

Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada

Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the correct functioning of the CFTR protein, the therapy’s developer announced. With a pill taken twice a day, Kalydeco, by Vertex Pharmaceuticals, makes it possible to resolve the underlying cause of cystic fibrosis (CF) in people with “gating” mutations — those resulting in the production of a faulty CFTR protein that have its channel’s gate stuck closed. The expansion of Kalydeco’s Market Authorization allows the use of this targeted therapy in young children with CF due to one of nine specific CFTR mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S

FDA puts Translate Bio’s rare disease mRNA drug on hold

The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a genetic disease that causes too much ammonia to build up in the blood. The hold comes just nine months after the agency lifted a clinical hold on a different program, its lead asset, in development to treat cystic fibrosis. The agency told Translate Bio verbally that it placed the hold because it had “additional clinical and nonclinical questions” after reviewing the IND for the candidate, MRT5201. The company submitted the application in December and is now is now waiting for an FDA letter with more details so it can work on the issues and get the treatment int

Featured Posts
Recent Posts
Archive
Search By Tags

Support Zahra's run for Palestine CF patients.

  • Facebook Clean
  • Twitter Clean
  • Instagram - Grey Circle
  • YouTube - Grey Circle
  • LinkedIn Clean