News

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

Nearly one-third of infants and toddlers with cystic fibrosis (CF) evaluated in a small study had low blood levels of zinc — a mineral whose insufficiency is associated with poor growth in otherwise healthy children. Still, the role of blood zinc levels “in growth and nutrition outcomes” for CF children…

A Phase 1/2 clinical trial of an aerosol gene therapy candidate, 4D-710, in people with cystic fibrosis (CF) has been cleared by U.S. Food and Drug Administration (FDA) and is expected to start this year, its developer, 4D Molecular Therapeutics, announced. The trial is designed as an open-label, dose-escalation…

Epicore Biosystems has launched in the U.S. a wearable patch allowing it to collect and analyze sweat for biomarker research, the company announced. Called the Discovery Patch Sweat Collection System, it may be useful in monitoring changes in sweat chloride in people with cystic fibrosis (CF), among other applications.

Students living with cystic fibrosis (CF) and attending or starting college, either at the undergraduate or graduate level, are invited to apply for one of 30 “Sacks for CF” scholarships. The annual program is run by the Boomer Esiason Foundation, a nonprofit dedicated to helping people with CF. Every…

Cystic fibrosis (CF) patients infected with the virus that causes COVID-19 are at a higher risk of hospitalization, a need for intensive care, and kidney injury relative to COVID-19 patients without this disease, according to a large, multi-center study in the U.S. While previous research reported that CF…

Cystic Fibrosis Canada has opened an initiative called Elevate, inviting people affected by cystic fibrosis (CF) to share their experiences, perspectives, and priorities with the nonprofit group.  “We have a history of asking for input from the community, but we felt…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Pharmacy benefits and services company Elixir has chosen Maxor Specialty Pharmacy to be its network’s exclusive provider of medications for cystic fibrosis (CF). Maxor Specialty specializes in rare diseases like CF and is one of four pharmacies in the U.S. to have access to all medications approved for cystic fibrosis,…

A research ethics committee in Australia has given Krystal Biotech the go-ahead to start a Phase 1 clinical study of KB407, the company’s investigational inhaled gene therapy for cystic fibrosis (CF). Enrollment for the trial is expected to start soon, according to Krystal, following approval from the…