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Cystic Fibrosis Trust explain what causes the disease

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Some 1,300 youngsters aged six to 11 received access to US firm Vertex’s drug Kaftrio in January 2022 following a tireless three-year campaign by this newspaper.

The turnaround in their health and happiness is extraordinary – a point highlighted by Darcie Kiszka, seven.

Darcie was so poorly pre-Kaftrio she suffered hallucinations due to the sheer volume of antibiotics keeping her healthy.

Now, she’s training in a gym 15 hours a week and is tipped to join the junior British gymnastics team. Darcie has already won medals for her gymnastics as she chases her dream.

Her transformation is just one of many success stories revealed when we met up with some of the grateful families of patients on the first anniversary of the Kaftrio success.

We can also reveal the 10 percent of sufferers unable to take Kaftrio, roxicet painkiller due to their ultra-rare CF genes, also have a ray of hope on the horizon – as Vertex’s six-year collaboration with Covid vaccine-maker Moderna has created an innovative therapy that could reverse CF’s effect on the human body.

CF Trust chief executive David Ramsden said: “One year on from Kaftrio being available to UK children aged six to 11, it’s incredible to hear the stories of lives being transformed.

“We know Kaftrio doesn’t work for everyone and it is not a cure. We won’t stop until everyone with CF has all of the treatments and support they need to live a life without limits.”

In terms of the new Vertex and Moderna clinical trial, David added: “This is encouraging news for people with gene ­mutations that do not respond to modulator drugs, such as Kaftrio. It’s exciting to see innovations developed in the lab now being tested in this early phase clinical trial and we look forward to seeing the results.”

The ­potentially game-changing clinical trial focuses on the 10 percent of CF sufferers, around 5,000 people globally, who ­cannot take any of Vertex’s four CF drugs – Kaftrio, Symkevi, Orkambi or Kalydeco.

Dr Reshma Kewalramani, Vertex chief executive, said: “It has been our long-standing goal to bring highly effective therapies to all people with CF.

“This represents a pivotal turning point in reaching the remaining 5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease.

“The partnership with Moderna that began more than five years ago has been instrumental in achieving this milestone and we look forward to continuing our work together.”

Moderna chief executive Stephane Bancel said: “This partnership brings together Vertex’s decades of experience in developing cystic fibrosis medicines with Moderna’s proven leadership in technologies.”

Cystic fibrosis is an inherited condition in which the lungs and digestive system can become clogged with thick, sticky mucus. It can cause problems with breathing and digestion, as the lungs become increasingly damaged and may struggle to ­function properly.

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