Note: This column describes the author’s own experiences with Trikafta (elexacaftor/tezacaftor/ivacaftor) and Orkambi (ivacaftor/lumacaftor). Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. Last Friday, I attended a Rare Disease Day event hosted by the National Institutes of Health (NIH)…

I lost my daughter, Jasmine, to cystic fibrosis (CF) at age 27, but we battled and lived with this rare genetic disease since before she turned 2 years old. In 1998, when Jasmine was 6, our mornings always started early. The kitchen was never quiet for long — it…

One year of treatment with Trikafta modestly eased some digestive symptoms and led to fewer reports of constipation in children with cystic fibrosis (CF), although those were already low before the triple-combination therapy, a new study reports. It also increased the proportion of patients with normal levels of fecal…

Five and a half years ago, I woke up from a double-lung transplant breathing as I hadn’t been able to in decades. Not just the volume or ease of it, but the absence of the fight that I’d become used to with cystic fibrosis (CF). The lungs just worked.

Lower insulin doses and less blood sugar variability were observed one year after adults with cystic fibrosis-related diabetes (CFRD) started Kaftrio. That’s according to results from the DIATRIM study (NCT06331000), a real-world, multicenter observational study conducted at 13 centers in France. Researchers retrospectively analyzed data from adults with…

I live with a rare disease. It seems strange to say that because it’s not so rare to me. Living with cystic fibrosis (CF) is the only life I’ve ever known. When I was born, researchers hadn’t yet discovered which gene was affected by CF or what chromosome…