By Niamh Shackleton
This adorable three-year-old needs to take nearly 30 tablets per day while her parents frantically beg strangers to help fund a ‘miracle’ drug from abroad.
Charlotte Robinson, from Rubery, Birmingham, suffers with cystic fibrosis and takes a cocktail of daily medication administered by her parents, Sophie, 33, and Aiden, 34.
But the family are now fundraising – and need about £17,000 – for a pioneering drug from Argentina as this will prevent the toddler’s lung function from declining in the future.
Sophie, a midwife, said: “We were heartbroken when Charlotte was diagnosed with cystic fibrosis, we knew the battle ahead we would have with her.
“There’s an American company who makes this ‘miracle’ drug, Orkambi, but charges around £104,000 a year per person – which is why it isn’t available through the NHS.
“However, an Argentinian company makes practically the same medication Lucaftor, for much, much cheaper, so it gives families of cystic fibrosis sufferers hope.
“Lucaftor would stop Charlotte’s lung function from declining meaning she would have a very good chance of a normal life; in addition to this, it would eventually reduce the amount of tablets she has to take a day.”
A yearly dosage of the drug for someone above the age of five is £23,000 – but with Charlotte only being three, she will only need three quarters of the average dosage making it around £17,000 to purchase.
Sophie added: “Charlotte has to take around 30 tablets per day to help with the condition and we have to do physio once or twice on a normal day too.
“If her coughing is particularly bad, we have to do physio three to five times a day – this was something we were taught to do by a professional to do when Charlotte came home from the hospital as a baby.
“This £23,000 drug would improve Charlotte’s quality of life massively.”
Charlotte was diagnosed with cystic fibrosis at just two weeks old after doctors conducted the heel prick test.
The blood spot screening involves taking a blood sample to find out if your baby has one of nine rare but serious health conditions – cystic fibrosis being one of them.
Sophie added: “When we were told about Charlotte’s diagnosis, we were informed that it’s the type of condition that’s incurable but ‘treatable’ – which is why she takes so many tablets a day.
“As a midwife, I used to be the one to inform parents if their child had something like cystic fibrosis and I’d reassure them that it would all be fine – but now I have a child myself who has it, I’m a lot more understanding of their concerns.
“It’s really difficult having a child with cystic fibrosis as you’re constantly worrying about them.
“If worst comes to worst and Charlotte’s lung function seriously declines, she would need constant breathing support and it would lower her life expectancy dramatically.
“A lot of people with the condition also require a lung transplant at some point in their life as their lung function drops so low.”
People with cystic fibrosis experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms such as fertility problems, lung infections and thinning of the bones.
The average life expectancy for those who were diagnosed at birth is 46 for men, and 41 for women.
In the hope of improving Charlotte’s quality of life, the Robinson family are fundraising for life changing medication for her.
They are hoping to be able to fund raise enough to purchase it in the new year.
To donate towards Charlotte’s miracle medication, visit: https://www.gofundme.com/f/2bt73a-helping-charlotte