Cystic Fibrosis

Orkambi Approved to Treat Younger Cystic Fibrosis Patients

By August 08, 2018

The approval was based on data from a 24-week, open-label, Phase 3 study in patients aged 2 to 5 years.

Novel Cystic Fibrosis Treatment Granted Fast Track Status

By April 04, 2018

Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808).

Cystic Fibrosis Combo Treatment Symdeko Gets FDA Approval

By February 13, 2018

Symdeko is Vertex's third medicine approved to treat the underlying cause of CF.

FDA Approves Kalydeco to Treat More Patients with Cystic Fibrosis

By August 01, 2017

Today's FDA approval follows the Agency's recent approval for 23 other residual function mutations based on in vitro data.

ACOG Updates Recommendations on Carrier Screening

February 27, 2017

Ethnic-specific, panethnic, and expanded carrier screening are all acceptable screening strategies.