Kaftrio, a drug used to combat cystic fibrosis symptoms, was hailed as “miracle” and “overwhelming” by one patient. The disease can cause terrible health problems and a shortened life expectancy
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A cystic fibrosis Patient has welcomed a new “miracle” drug in her battle with the disease.
Gillian Docherty, 36, has struggled with the disease, but described the effects of a new drugKaftrio, on her life as “amazing”.
have people with the hereditary disease a much shorter life expectancy than average, due to how severely the condition can affect people.
Experts hope that the new drug will continue to contribute to a better quality of life for patients suffering from it.
What is cystic fibrosis and what do we know about a new drug that may help?
What is cystic fibrosis?
Cystic fibrosis is an inherited disorder that “results in the build-up of sticky mucus in the lungs and digestive system.”
The genetic problem is caused by a faulty gene that affects the movement of salt and water in and out of cells.
The after-effects of this cause serious health problems such as lung infections and digestive disorders.
Symptoms usually begin in early childhood and babies are usually diagnosed soon after they are born.
Constant pressure on the lungs and digestive system causes these organs to become damaged over time.
Symptoms of cystic fibrosis include:
- shortness of breath
- diarrhea or constipation
- Difficulty gaining weight or growing
That NHS said: “Cystic fibrosis gets worse over time and can be fatal if it leads to a serious infection or if the lungs stop working properly.
“But people with cystic fibrosis are now living longer because of advances in treatment.”
Can cystic fibrosis be cured?
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There is still no cure for cystic fibrosis, but there are promising signs of a new drug being developed to combat its effects.
A newly approved drug called Kaftrio, which was first made available to UK patients in August 2020, appears to be helping.
Gillian had previously sorted her end-of-life affairs for her husband Andy, but within two weeks of taking the drug her lung function went from 38% to 74%. It is now over 80%.
The recovery brought happy news as she went from being “unable to climb the stairs” to giving birth to a baby boy in December last year.
She said: “I went to sleep that first night and woke up seven hours later – something that hadn’t happened in years.
“Midnight bouts of coughing and waking up with chest pains were the norm for me.
“That first morning I slept through the night, Andy and I just woke up and looked at each other, speechless at how effective the drug was.”
dr Gordon Magregor is a consultant doctor at Queen Elizabeth University Hospital (QEUH) in Glasgow and one of the staff taking care of Ms Docherty.
He said: “Kaftrio has truly been a game changer in treating the patients it is clinically appropriate for.
“It helps keep them away from the hospital and allows them to lead fairly normal lives, although their condition will always require some level of care from staff.”
https://www.mirror.co.uk/lifestyle/health/cystic-fibrosis-symptoms-explained-patient-26737375 Cystic Fibrosis symptoms explained while patient praises drug Kaftrio