Patricia Inácio, PhD, science writer —

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Routine evaluations of how cystic fibrosis (CF) patients and their families are coping with the burden of therapeutic regimens — usually daily therapies that are complex and time-consuming — are an important tool to enhance treatment adherence and create a strong working relationship between patient and care provider, a…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Respinova’s non-invasive Pulsehaler, a medical device meant to ease breathing in patients with airway restrictions, such as in cystic fibrosis (CF), has been cleared for marketing in the U.S. Pulsehaler was granted 510(k) clearance from the U.S. Food and Drug Administration (FDA) following the company’s submission of a…

A Phase 2 clinical trial assessing the safety and efficacy of BX004 — BiomX’s phage therapy candidate for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF) — remains on track to have results by the end of this year, the company announced. While this timeline had been…

Specific immune cells called macrophages in cystic fibrosis (CF) are unable to kill the bacteria Burkholderia cenocepacia due to impairments in mitochondria, the cell’s powerhouses, a new mouse study says. The finding suggests that boosting mitochondria function could help resolve chronic infection in CF. The study “Defective immunometabolism…

The Cystic Fibrosis Foundation (CFF) awarded up to $3.75 million to Matinas BioPharma to support preclinical studies of MAT2501, a potential oral antibiotic to treat nontuberculous mycobacterium (NTM) infections in people with cystic fibrosis (CF) and other lung diseases. A goal of this early research work is…

New findings from animal studies support the safety and effectiveness of KB407, a potential inhaled gene therapy for cystic fibrosis, its developer, Krystal Biotech, announced. Preclinical work is ongoing, and the therapy is expected to enter clinical testing in patients by mid-2021. “I am particularly encouraged by progress with KB407 for…

Patient enrollment and dosing has resumed in a Phase 1/2 clinical trial testing MRT5005, Translate Bio‘s potential messenger RNA (mRNA) therapy for people with cystic fibrosis (CF) regardless of their underlying mutation. The trial, called RESTORE-CF trial (NCT03375047) and enrolling up to 40 adults with stable disease at 17 centers…

The Cystic Fibrosis Foundation (CFF) has awarded funding to enGene to support its work on a non-viral and inhalable gene therapy that could deliver a working copy of the CFTR gene to the lungs of people with cystic fibrosis (CF). “Today’s announcement reflects yet another milestone on our path…