Understanding the structure of the cystic fibrosis transmembrane regulator (CFTR) and the effect of mutations on its function is a crucial part in the development of new drugs that tackle the basic defect.
This week the devolved governments in Wales and Scotland announced details of spending plans around cost of living payments, benefits, and national living wage. Here we look at what it could mean for people living with CF in Wales and Scotland.
Vertex Pharmaceuticals have announcedthat they have approval from the Food and Drug Administration (FDA) to begin clinical trials of their ‘mRNA’ genetic therapy as a treatment for cystic fibrosis. This trial is the result of a collaboration between Vertex and Moderna.
In 2021/22, our dedicated Helpline team responded to over 4,200 enquiries from the CF community on a huge range of topics, from coping with a new diagnosis to supporting you through the COVID-19 pandemic. During what was another tough year for everybody, our Helpline service continued to adapt to the changing needs of our community, providing support, information and signposting when people with CF and their families needed it most.
Cystic Fibrosis Trust is today launching an urgent Festive appeal to help people with cystic fibrosis who are in desperate need this winter due to the impact of the rising cost of living.
Finding ways to reduce and simplify treatment burden has been chosen by the CF community as a top research priority. Research studies are underway to find out if it is safe to reduce or remove muco-active treatments for people with CF who are taking Kaftrio. One of these studies is the CF STORM study in the UK and another is the SIMPLIFY study in the US. As the results of SIMPLFY have just been announced, we explain why these studies are so important, and how you can take part in CF STORM.
