News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

In 1960, a 19-year-old woman with cystic fibrosis (CF) made medical history when she gave birth after only 34 weeks of pregnancy. The patient died three months after delivery. In 1986, by which time the life expectancy of people with the disease had risen substantially, some 50 women with…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Liver ultrasounds can help identify children with cystic fibrosis (CF) who are at higher risk of developing liver disease, a new study shows. The study, “Heterogeneous Liver on Research Ultrasound Identifies Children with Cystic Fibrosis at High Risk of Advanced Liver Disease: Interim Results of a…

AB569 is safe and effective at killing bacteria resistant to traditional antibiotics, in particular Pseudomonas aeruginosa (P. aeruginosa), an important disease-exacerbating microbe in cystic fibrosis (CF), a study showed. The study, “AB569, a nontoxic chemical tandem that kills major human pathogenic bacteria,” was published in the journal…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for cystic fibrosis (CF), the therapy’s developer, Translate Bio, has announced. Fast track is given to medications that aim to treat serious or life-threatening conditions and…

Enrollment is complete for a European initiative to test an organoid-based, personalized-medicine approach to treating cystic fibrosis, with the first trial starting later this year, said Proteostasis Therapeutics (PTI) and CF Europe. The Human Individualized Therapy of CF (HIT-CF) initiative will test PTI drug combinations through…

Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported.

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…