Forest Ray PhD,  —

AzurRx BioPharma has enrolled the first three patients in its Phase 2b trial investigating MS1819 as a treatment for exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients. The trial, dubbed OPTION 2 (NCT04375878), aims to enroll 30 CF patients ages 18 and older. Top-line…

Cystic Fibrosis Ireland has set aside up to €120,000 ($141,150) to provide personal protective equipment (PPE) to people with cystic fibrosis (CF) in the Republic of Ireland. Those interested may apply for individual grants of €100 per applicant through Aug. 22. Applications to the PPE grant should be…

The government of  Northern Ireland reached a deal with Vertex Pharmaceuticals to make Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for cystic fibrosis (CF) patients, once the medication receives its European license. The agreement follows similar ones that the company made recently with England and Wales.

A new technology could enable faster and more accurate cystic fibrosis (CF) diagnoses by non-invasively tracking the movement of air through even the fine structures of the lungs at high-resolution. A study done in mice showed this technology, called X-ray velocimetry (XV), could pinpoint localized areas of abnormal airflow…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Cystic fibrosis (CF) patients who monitored their lung function at home using NuvoAir‘s handheld spirometer and smartphone app were able to better engage in telemedicine, a U.K. survey found, with these patients reporting a more than 30% drop in their number of scheduled and urgent in-person consultations. The study based…

Anxiety related to fears of COVID-19 infection was higher among mothers of children with cystic fibrosis (CF) than those of healthy children, although teenage patients themselves were less anxious than age-matched peers without CF, a survey of families in Turkey reported. Mothers of young patients with chronic lung infections, and…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Thousands of people in England with cystic fibrosis (CF) soon will be equipped with digital home spirometry kits. This will allow patients age 6 and older to monitor and share vital lung function information with their physicians without leaving home unnecessarily to make often-lengthy journeys to the hospital. Monitoring…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.