Author Archives: Larry Luxner

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…

Two years ago, Israel became only the fourth country in the world — after the U.S., Germany, and Belgium — to agree to underwrite the cost of Vertex Pharmaceuticals’ three available therapies for cystic fibrosis: Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combo). But that’s not…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Tiffany Rich is something of a celebrity in the world of cystic fibrosis (CF). Diagnosed at birth, she graduated college while awaiting a double lung transplant. The co-founder of Salty Cysters, she’s been featured in Cosmopolitan magazine, and has written numerous blogs on the disease. Recently, the 28-year-old…

Michael Boyle, MD, has laid out a five-year strategic plan for the Cystic Fibrosis Foundation (CFF), following his Jan. 1 appointment as president and CEO of the Maryland-based charity. In a three-minute video message posted Jan. 9 to the CFF website, Boyle said the 2020-24 plan will focus…

People with cystic fibrosis (CF), like their healthy counterparts, often neglect sleep. But that can lead to problems down the road, warned three CF experts at the 2019 North American Cystic Fibrosis Conference in Nashville. More than half of all CF patients have sleep complaints, said pulmonologist Eliot…

Oregon retiree Carol Birch, 59, considers herself especially lucky to have made it this far in life. “I’ve never felt that I fit the typical role of someone with cystic fibrosis (CF),” Birch told attendees at the 2019 North American Cystic Fibrosis Conference (NACFC) in Nashville, Tennessee. Diagnosed at…

Preston W. Campbell III, MD, recently bid farewell as president and CEO of the Cystic Fibrosis Foundation (CFF) in Nashville — the same city where he began his long association with the disease 30 years ago as a pediatric pulmonologist at Vanderbilt University’s Cystic Fibrosis (CF) Care Center.

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.