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 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…
Among people with cystic fibrosis (CF), antibiotic-resistant Staphylococcus aureus is sometimes transmitted among relatives, but rarely in healthcare settings, an analysis of patients at a CF center in Iowa suggested. Findings also indicate that distinct subsets of S. aureus may be associated with differences in disease progression. These results were in the…
This year’s Cystic Fibrosis Foundation (CFF) Impact Grants will support three new virtual programs that support the well-being of people of all ages living with cystic fibrosis (CF). These winning projects range from a virtual video performance with tailored songs for hospitalized children with CF, to online exercise classes for teenagers,…
Most primary care providers (PCPs) in Ontario — family physicians, pediatricians, and midwives — voiced a willingness and confidence in providing some level of care to infants testing positive for cystic fibrosis (CF) in newborn screening programs, a Canadian survey reported. A majority favored treating minor illnesses like infections…
Diagnostic odysseys are common for people with rare diseases. But for Terry Wright, it took 54 years of gastrointestinal pain, lungs clogged with mucus, countless emergency room trips to treat pneumonia, and surgeries to get to the bottom of a disease that should have been clear from the beginning:…
Assessment, documentation, and education regarding cannabis and cannabidiol (CBD) use among cystic fibrosis (CF) patients varies across centers in the U.S., according to a survey of nearly 300 CF healthcare providers. This variability may be related partially to the different laws between U.S. states regarding these controversial substances, the…
SPL84-23, an investigational antisense oligonucleotide (ASO), restored the levels and function of CFTR — the defective protein in cystic fibrosis (CF) — in lab-grown cells from patients carrying a common and disease-causing splicing mutation, a study reported. Based on these positive preclinical findings, SpliSense plans to launch a Phase 1/2…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
A novel compound that may hold promise for treating the roughly 11% of cases of cystic fibrosis (CF) that are caused by nonsense mutations has been described in a new study. The study, “A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1…
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