February 15, 2018
The Food and Drug Administration approved a new drug that helps address the underlying cause of cystic fibrosis, not simply its symptoms. The drug, a combination of tezacaftor/ivacaftor, is for patients aged 12 and older with certain cystic fibrosis mutations.
May 30, 2017
Orphan drugs aren’t always reaching the patients who need them, and an advocacy group known as the Cystic Fibrosis Engagement Network has a few ideas as to why.
April 18, 2017
Promising clinical trials data has excited patients with cystic fibrosis and their families. But will access barriers dash their hopes for more and better treatment options?
February 28, 2017
Rare Disease Day brings international attention to the plight of patients who struggle with ALS, Gaucher disease, cystic fibrosis and other less common conditions. This year’s theme is research, a challenge for any disease state but especially for conditions with small patient populations.
February 23, 2017
As Congress continues to debate the future of the Affordable Care Act, a new advocacy group has a message for key legislators: protect cystic fibrosis patients as you reshape health care policy.
November 3, 2016
More than a year after a breakthrough drug for cystic fibrosis became available, fewer than half of patients who meet the Food and Drug Administration’s criteria can actually access the medication.