Charities unite to help children with cystic fibrosis
Dr Rebecca Dobra at the National Heart and Lung Institute, Imperial College London has been awarded funding to investigate long term health outcomes for children with designation of CF screen positive, inconclusive diagnosis (CFSPID). UCL-based Dr Guy Moss has been awarded funding to investigate whether medicines called potassium channel activators could be used as new treatments for CF in the future.
Predicting long-term health outcomes for children with an inconclusive diagnosis
Newborn screening helps detect CF early, but some babies receive inconclusive results, creating uncertainty about their future health. The research team is aiming to identify ways to predict which of these children will remain healthy, and which may later develop CF or a related condition. The long-term goal is to enable doctors to provide tailored care for each child, reducing parental anxiety, avoiding unnecessary monitoring of healthy children, and enabling early intervention for those most at risk of developing disease.
Could potassium channel activators be a potential treatment for CF?
Dr Moss and colleagues will conduct lab studies to find out whether potassium channel activators can improve the effectiveness of CFTR modulators. They will also investigate whether new combinations of potassium channel activators and other medicines might be an effective way to treat people who are not able to benefit from CFTR modulators.
Our partnership with Action Medical Research has played a vital role in driving forward the breakthroughs that will lead to the new treatments and support that families and children affected by CF urgently need. We won’t stop until everyone can live without limits imposed by CF.
Dr Lucy Allen, Director of Research and Healthcare Data at Cystic Fibrosis Trust
Investing together in this important area can really help move us closer to developing much-needed new approaches and treatments to help tackle cystic fibrosis in children.
Sarah Wagstaffe-Jones, Director of Research at Action Medical Research
CF continues to make too many lives too tough and too short, and Cystic Fibrosis Trust will not stop until everyone with CF can live a life that’s not limited by their condition. To help Cystic Fibrosis Trust fund more important work, click here.
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