A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…
News
For children with cystic fibrosis (CF), newborn screening is associated with markedly improved real-world outcomes, including earlier diagnosis, greater weight and height gains, and fewer bacterial infections, new research demonstrates. In fact, infants are now diagnosed with the genetic disease at a median of 0 months…
The Cystic Fibrosis Foundation (CFF) is partnering with Pioneering Medicines, an initiative of Flagship Pioneering, to support the development of various types of gene therapy for cystic fibrosis (CF). The foundation is ready to invest up to $110 million to move therapy candidates into clinical testing, starting with an…
People with cystic fibrosis (CF) who start on the triple therapy Trikafta may be at greater risk of problems associated with excessive weight or obesity, such as metabolic syndrome — a group of disorders known to raise a risk of heart, liver, and kidney disease. “With the approval…
Treatment with Orkambi (lumacaftor/ivacaftor) and/or Symdeko (tezacaftor/ivacaftor) may lessen liver damage in children and adolescents with cystic fibrosis (CF) who have liver involvement, new data indicate. “We detected improvements in validated noninvasive biomarkers of liver fibrosis [scarring] and cirrhosis in children and adolescents treated with” these therapies, the…
Treatment with Trikafta can help people with cystic fibrosis (CF) to put on weight, according to new data from a small clinical trial. These findings indicate that traditional guidelines for CF nutrition — which urge a…
Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD,…
Since the approval of Trikafta in the U.S., Nationwide Children’s Hospital in Ohio has seen a steep increase in the number of women with cystic fibrosis (CF) choosing to become pregnant. “Due to the large pregnancy volume we…
Aceragen reached an agreement to acquire Arrevus, including its late-stage clinical program of ARV-1801 — now called ACG-721 — an experimental oral therapy for pulmonary exacerbations in cystic fibrosis (CF) patients. Aceragen is planning to launch a Phase 2/3 clinical trial in the second half of 2022…
The Cystic Fibrosis (CF) Foundation has invested $3 million in Armata Pharmaceuticals to support development of AP-PA02, a bacteriophage now being evaluated in a clinical trial for its ability to treat respiratory infections caused by Pseudomonas aeruginosa in people with cystic fibrosis (CF). “We are pleased by…
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