The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
News
Cystic fibrosis (CF) patients showed improved clinical outcomes and less disease progression after long-term treatment with Kalydeco (ivacaftor), according to an observational study. Researchers found that Kalydeco-treated CF patients had a significantly lower risk of death, pulmonary exacerbation, hospitalization, transplant, and prevalence of CF-related complications and pathological microorganisms,…
Tobramycin, an antibiotic often used to treat acute lung infections in cystic fibrosis patients, is safe for once-a-day use in children under age 6, with findings of minimal kidney and no inner ear toxicity in these pediatric patients, a study reports. The research, “Safety of Extended Interval Tobramycin…
Patients with cystic fibrosis (CF) are at higher-than-average risk of developing gastrointestinal cancers, especially those who underwent a lung transplant, a new study shows. Transplant patients with CF were found in this retrospective study to have a five-times greater risk of gastrointestinal cancer than those who have not had a…
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…
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