Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada
Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the...
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...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
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...
#NACFC2018 — Vertex Presents Latest Clinical Data on CFTR Modulators
Vertex Pharmaceuticals’ VX-659 or VX-445 in combination with Kalydeco(ivacaftor) and tezacaftor (VX-661) can improve the lung function of...
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...
FDA Expands Kalydeco’s Approval to 1-year-old Toddlers with Cystic Fibrosis
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) as the first-ever therapy to treat the underlying cause of...
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...
Vertex, NHS England no closer to Orkambi settlement
Vertex and NHS England remain locked in a battle over the provision of cystic fibrosis drug Orkambi, seemingly no closer to a settlement...
