FDA Approves Symdeko for Treatment of CF Children, Ages 6 to 11, with Certain CFTR Mutations
The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of...
Symdeko Approved in Australia for CF Patients Age 12 or Older With Certain Gene Mutations
Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy...
FDA puts Translate Bio’s rare disease mRNA drug on hold
The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a...
The Bittersweet Downside of CFTR Modulators
I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three...
Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities
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...
Vertex and Treating CF: 3 Disease-modifying Therapies Make Leap from Lab to Patients
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...
Vertex and Treating CF: Stepping Out on Long Road to ‘Medical History’
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...
Vertex Anticipates Completing Enrollment of Phase 3 Trials Testing Triple Combo Therapies
Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat...
Health Canada Approves Symdeko to Treat CF Patients 12 and Up with Certain Gene Mutations
People ages 12 and older with cystic fibrosis (CF) who live in Canada can now be treated with Vertex Pharmaceuticals’ Symdeko...
