ORKAMBI IS FDA APPROVED!
THIS IS what your money is doing for those living with Cystic Fibrosis! The FDA approved a combination drug (lumacaftor/ivacaftor – Orkambi™) – on July 2! Orkambi targets the underlying cause of the disease in people with two copies of the most common CF gene mutation. Nearly half of the individuals with CF have two copies of this mutation and could eventually benefit from the therapy. The drug which will help Katie (she has one copy of the main CF gene and one unidentified mutation) is in phase three trials now and will hopefully be on the market in the next 2 to 3 years!!! We are fighting harder than ever to help all those living with CF. We will NOT STOP running, riding or doing whatever it takes to WIPE OUT CF!