News

A team of professional cyclists are riding six custom-designed and hand-painted bicycles — each inspired by a child whose medical needs were supported by the UnitedHealthcare Children’s Foundation (UHCCF)  — at the 2018 Amgen Tour of California now underway. The Rally Cycling team’s goal is to help raise money…

An advisory committee composed by experts in the field of cystic fibrosis will help to guide the design of the global Phase 3 program testing PTI-428, the company developing the potential treatment, Proteostasis Therapeutics, announced. Its formation is a key step in ensuring that the program follows regulatory requirements, while retaining…

The approved CFTR modulators developed by Vertex Pharmaceuticals are too expensive for the clinical benefit they can provide to cystic fibrosis (CF) patients, according to the Institute for Clinical and Economic Review (ICER). ICER evaluated the effectiveness and costs of Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and…

Cystic Fibrosis Canada (CF Canada) launched a national campaign in May, which is Cystic Fibrosis Awareness Month, to call on the government to make life-changing medications accessible to those living with (CF). Orkambi (ivacaftor/lumacaftor), developed by Vertex Pharmaceuticals, was approved in the United States in 2015 for…

The Cystic Fibrosis Foundation will award up to $11 million to Icagen to support a multi-year research-program aiming to develop new therapies for patients with cystic fibrosis (CF) caused by nonsense mutations, who may fail to respond to the current CFTR modulator therapies. The aim of the research is to…

When the Cystic Fibrosis Foundation (CFF) was established in 1955, most people with cystic fibrosis (CF) didn’t make it to their sixth birthday. Today, the average life expectancy of a CF patient is 47 years. To date, the U.S. Food and Drug Administration has approved 12 CF therapies. Three of…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Vertex Pharmaceuticals recently awarded $400,000 in scholarships for people living with cystic fibrosis (CF) and their family members, as part of its second “All in for CF” scholarship program. The program awarded 80 scholarships worth $5,000 each, to allow more people to pursue higher education in the…

AllianceRx Walgreens Prime announced that it has access to a series of limited-distribution drugs (LDDs) for its specialty patients, including the recently approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis (CF). Other medications available include Austedo (deutetrabenazine) for Huntington’s disease, Fasenra (benralizumab) for severe asthma, and Hemlibra…

Vertex Pharmaceuticals is launching Phase 3 clinical trials to evaluate the safety and effectiveness of its investigational therapy, VX-445, in combination with VX-661 (tezacaftor) and Kalydeco (ivacaftor) for cystic fibrosis (CF). The AURORA program will consist of two randomized, double-blind Phase 3 trials — AURORA F/MF…