Larry Luxner,  —

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

When Shira Zagury joined the Cystic Fibrosis Foundation of Israel as CEO eight years ago, only one cystic fibrosis (CF) patient sat on the nine-member board of directors. Today, five of the nine are adults with CF — a reflection of the ever-increasing life expectancy of those born with the disease.

Cystic fibrosis (CF), once an exclusively pediatric disease, is clearly one no longer. Of the 32,000 or so Europeans registered with CF, nearly half are more than 18 years old, and babies born with the illness today can expect to live into their 50s and 60s. This means issues like…

Exercise clearly benefits kids with cystic fibrosis (CF), yet these children often lack the motivation to swim, run, box, play football or lift weights. A British healthcare conglomerate says it has found a way to get these kids moving — at no cost to them or their families. Nuffield…

Anxiety and depression among cystic fibrosis (CF) patients — and their parents — is far more common than most people realize, according to a panel of experts at the recent 41st European Cystic Fibrosis Conference (ECFS) in Belgrade, Serbia. The discussion featured eight mental health experts, and focused on…

SPX-101, designed to help clear mucus from the lungs of people with cystic fibrosis, has the potential to treat all CF patients — regardless of disease-causing mutations. The experimental peptide, which blocks the overactive sodium channels that line CF lung cells so more fluids are retained on airway surfaces to hydrate mucus,…

Some 318,000 health-related software apps are now on the market, with another 200 appearing every day. Yet a British cystic fibrosis expert questions how effective they are at managing lung disease, for reasons that range from the needless anxiety they can cause to sporadic use and concerns with patient data protection.

Europe’s largest annual gathering of cystic fibrosis (CF) experts ended June 9, capping four days of speeches, workshops, and poster presentations that attracted 1,832 participants from 56 countries. The largest contingents at the 41st European Cystic Fibrosis Conference, held in Belgrade, Serbia, came from the United Kingdom (304) and Serbia itself…

The European Cystic Fibrosis Society presented three scientists — one Portuguese, one Mexican, and one German — with the organization’s 2018 “Young Investigators Awards” on June 9, the last day of its 41st European Cystic Fibrosis Conference (ECFS) in Belgrade, Serbia. The winners are Iris Silva, Samuel Lara Reyna, and…

Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…