News

Student Appointed to Ohio Rare Disease Advisory Council

Andrea Hoffman, an Ohio Northern University (ONU) student who also is a cystic fibrosis (CF) patient and advocate, has been appointed by Ohio Gov. Mike DeWine to the Ohio Rare Disease Advisory Council, according to a university press release. In her two-year term, Hoffman, an ONU junior, will…

Depression, Anxiety Common Among CF Patients

Depression and anxiety are common among people with cystic fibrosis (CF), a systematic review reports. The study “Global Burden of Anxiety and Depression among Cystic Fibrosis Patient: Systematic Review and Meta-Analysis” was published in the International Journal of Chronic Diseases. In people with CF, a genetic defect…

Patients Report Satisfaction, Concerns With Opioids

People with cystic fibrosis (CF) who use opioids to manage pain commonly report being satisfied with the treatment, but also have concerns about the potential for addiction and stigmatization, according to the results of a recent patient survey. The findings highlight “the need for guideline-driven practice standards, including the…

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

Vertex Planning Phase 3 Trials in New Triple Combo Therapy for CF

Vertex Pharmaceuticals is initiating two Phase 3 clinical trials into the safety and efficacy of a potentially new, next-generation triple combination therapy for cystic fibrosis (CF): VX-121 plus tezacaftor and VX-561 (deutivacaftor). Expected to begin later this year, the development program will compare this once-daily therapy with Vertex’s…