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Daily Mirror
Daily Mirror
Lifestyle
Benjamin Lynch

Cystic fibrosis symptoms explained as patient hails new 'miracle' drug Kaftrio

A cystic fibrosis patient has hailed a new 'miracle' drug in her fight against the disease.

Gillian Docherty, 36, has struggled with the disease but described the effects of a new drug, Kaftrio, on her life as "mind-blowing".

People with the hereditary disease have a much shorter life expectancy than average, due to how badly the condition can affect people.

Experts are hopeful the new drug will continue to add to a better quality of life for patients suffering from it.

So what is cystic fibrosis and what do we know about a new drug that is able to help?

What is cystic fibrosis?

Cystic fibrosis symptoms cause a shorter life expectancy for sufferers (Cystic Fibrosis Trust/Marcel Reinard/PA Wire)

Cystic fibrosis is a hereditary disease that "causes sticky mucus to build up in the lungs and digestive system".

The genetic problem is caused by a faulty gene that impacts on the movement of salt and water in and out of cells.

The knock-on effects of these cause severe health problems such as lung infections and trouble digesting food.

Symptoms usually start in early childhood and babies tend to be diagnosed soon after they're born.

Constant pressure on the lungs and digestive system mean that over time, these organs become damaged.

Symptoms of cystic fibrosis include:

  • Wheezing
  • Shortness of breath
  • Jaundice
  • Coughing
  • Diarrhoea or constipation
  • Difficulty putting on weight or growing

The NHS said: "Cystic fibrosis tends to get worse over time and can be fatal if it leads to a serious infection or the lungs stop working properly.

"But people with cystic fibrosis are now living for longer because of advancements in treatment."

Can cystic fibrosis be cured?

Gillian Docherty and her husband Andy were able to welcome baby Frederick after Gillian was treated with Kaftrio (UGC)

Cystic fibrosis cannot yet be cured, but there are promising signs in a new drug designed to tackle its effects.

A newly approved drug known as Kaftrio, first made available to UK patients in August 2020, seems to be helping.

Gillian had previously been sorting her end of life affairs for her husband Andy, but within two weeks of using the drug, she saw her lung function increase from 38% to 74%. It is now above 80%.

The recovery brought happy news as she went from being "unable to climb the stairs" to having a baby boy in December last year.

She said: "That first night, I went to sleep and woke up seven hours later – something that hadn’t happened for years.

"Midnight coughing fits and waking up with chest pain were the norm for me.

"That first morning I slept through the night Andy and I just woke up and looked at each other, lost for words at how effective the drug was."

Dr Gordon Magregor is a consultant physician at the Queen Elizabeth University Hospital (QEUH) in Glasgow and one of the team looking after Mrs Docherty.

He said: "Kaftrio has really been a gamechanger in treating the patients for whom it is clinically appropriate.

"It helps keep them out of the hospital and enables them to live pretty normal lives, albeit they will always need a level of care from staff for their condition."

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