Emma Yasinski,  —

Waiting for a lung transplant in the intensive care unit at a New York hospital in November 2017, a 21-year-old pulmonary hypertension patient named Miriam Holman sued the U.S. Department of Health and Human Services. Her lawsuit accused the department of allocating organs based on a patient’s geographic location,…

Vertex Pharmaceuticals announced it plans to request by year’s end that the label for Symdeko (tezacaftor/ivacaftor and ivacaftor), its recently approved disease-modifying therapy for cystic fibrosis (CF), be expanded to include children as young as 6 years old, the company announced in a recent investor report. It also plans to…

Editor’s note: This is the last of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at treatment pricing disputes, and the company’s support for the CF community. Vertex is well-known for its three approved cystic fibrosis (CF) treatments —…

Editor’s note: This is third in a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at potential “next-generation” treatments that range from triple combinations in or readying for clinical trials, to gene editing that has a way to go. The research that led…

Editor’s Note: This is the second of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we detail the steps that made three disease-modifying oral treatments for cystic fibrosis possible. Kalydeco (ivacaftor), as Vertex‘s cystic fibrosis potentiator came to be known, was a small…

Editor’s note: This is the first of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. We begin by recounting the discoveries that made three disease-modifying treatments for cystic fibrosis — by the same science team — possible in a matter of years.  To the interested…

Five scientists whose work has been instrumental in developing disease-modifying treatments for cystic fibrosis (CF) are recipients of the 2018 Warren Alpert Foundation Prize. Until recently, therapies for CF targeted only the symptoms of the disease. The U.S. Food and Drug Administration (FDA) approved the first disease-modifying therapy, Kalydeco (ivacaftor, marketed…