Delivering nutrients through dialysis could help people with co-occurring cystic fibrosis (CF) and kidney disease meet their nutritional requirements, a recent case report illustrates. The report, “Intradialytic parenteral nutrition improves nutritional status in a complex cystic fibrosis patient with redo double lung transplant and end-stage renal…
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Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized triple combination for cystic fibrosis (CF) patients without available treatment options, from the Dutch Medicines Evaluation Board. Regulatory officials outlined necessary steps in this planned, pivotal trial that would support…
University students with cystic fibrosis (CF) are invited to apply to AbbVie’s CF Scholarship program and a chance to receive up to $25,000 for the upcoming school year. The program supports U.S. residents with CF…
Long-term treatment with Kalydeco (ivacaftor) in people with cystic fibrosis (CF) is associated with a better body mass index, lung function, and a lesser effort required to breath, a small study reports. The single-site study, “Long‐term…
Most healthcare professionals are not fully prepared for cystic fibrosis (CF) end-of-life care, and further training and coordination between CF and palliative care teams would be helpful, according to a small, single-center survey in the U.K. Its researchers recommended a broader survey — one nationwide, or even international — to…
An unusual clinical presentation can hinder proper diagnosis for people with cystic fibrosis (CF), even with newborn screening, a new case report contends. The report, “Uncommon clinical presentation of cystic fibrosis in a patient homozygous for a rare CFTR mutation: a case report,” was published in…
When it comes to lung transplants, a study has found that the practice of many cystic fibrosis (CF) specialists differs from that of updated recommendations, highlighting a need to educate providers on new guidelines. The study, “Lung transplant referral practice patterns: a survey of cystic fibrosis physicians and general…
Savara Pharmaceuticals has stopped patient enrollment into two of its ongoing clinical trials in cystic fibrosis (CF) patients — AVAIL and ENCORE — for safety reasons due to the COVID-19 pandemic. Steps will also be taken to ensure that those already enrolled in both studies continue…
ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function…
Translate Bio‘s therapy MRT5005 has been given a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis in children, while its ongoing clinical trial investigating the therapy in adult patients faces delays due to the COVID-19…
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