The European Medicines Agency (EMA) announced today that it has recommended the approval of the CF drug Kalydeco™ for people with the G551D mutation ages 6 and older in the European Union.
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On Aug. 27, leaders from the Cystic Fibrosis Foundation, along with several people from the CF community, participated in a virtual public meeting hosted by the Institute for Clinical and Economic Review to provide insights and expertise on the value of CFTR modulators.
You can best manage your health when you know what to expect, what resources you can use, and a community of people with whom you can share your experiences.
Estamos buscando tratamientos potenciales para las personas con FQ que tienen mutaciones raras y sin sentido. Es posible que no aprovechen de los moduladores, que corrigen la proteína reguladora de la conductancia transmembrana de la fibrosis quística (CFTR, por sus siglas en inglés) que funciona mal.
For so long, my identify was defined by beating the odds and achieving a successful career despite having CF. But recently, health complications have forced me to create a new identity focused on self-care. With the help of my support system, I am slowly adapting to this new chapter.
I was recently reminded of what my life used to be like before Trikafta® — and I immediately felt guilty for forgetting. But maybe it’s OK to let go of the past and embrace a future of hope.
Theratyping matches therapies, or medications, to specific types of mutations. The primary goal of theratyping is to identify which mutations respond to certain CFTR modulators, thereby helping people with rare CFTR mutations gain access to already approved modulators quickly and safely.