MP Marie Rimmer has praised drug firms for lowering prices on a life changing drug but says that while she understands "that companies need profits, there is nothing more valuable than a child’s life".
Kaycee Bradshaw, who is the youngest of six siblings from Sutton, was diagnosed with Batten disease in 2016.
Children with the condition rarely survive past the age of 12. The disease causes patients to have seizures, muscle spasms, progressive dementia, visual impairment leading to blindness. It also results in the loss of speech and mobility.
Kaycee is currently one of five children receiving an enzyme replacement treatment called cerliponase alfa, also known as Brineura, which slows the effects of the incurable condition.
Happy Kaycee
Brineura can slow the progression of the disease and some children like Kaycee have taken part in a trial or are being given the drug on compassionate grounds by pharmaceutical manufacturer BioMarin.
But this was under threat as treatment cost more than £500,000 per patient, so the NHS and NICE – the National Institute for Health and Care Excellence labelled it "too costly".
However, parents, such as Kaycee's mum Claire Riley, did not back down and alongside their MPs and advocates, tirelessly campaigned for treatment costs to be lowered to save their children's lives
On Wednesday, September 11 it was announced that after years of battle, the NHS has successfully struck a deal with manufacturers to provide the treatment.
Read what her mum had to say here.
Claire celebrating with Kaycee
St Helens South and Whiston MP Marie Rimmer said: "It is fantastic news that all the children who suffer from Battens disease, including Kaycee Bradshaw in my constituency, will finally have easy access to this drug that will so greatly enhance their quality of life.
"I commend Biomarin for coming back to the negotiation table and offering the drug to the NHS at a fair price.
"While I understand that companies need profits, there is nothing more valuable than a child’s life.
"I also hope Biomarin will take a similar approach with other life changing drugs that are currently unavailable to NHS England, due to high costs."
Biomarin is also the drug manufacturer who makes the drug Kuvan which would see those living with PKU take a pill each day and allow them to have a "normal" diet without many restrictions.
PKU (phenylketonuria) is a condition where sufferers are born unable to process protein.
Kaycee
PKU is caused by the deficiency of an enzyme called phenylalanine, the absence of which causes phenylalanine to accumulate in the blood, which can result in brain damage.
Kuvan, is available in other countries but not available on the NHS – which instead recommends a strict diet and medication.
The National Society of Phenylketonuria has been campaigning for the NHS and Kuvan supplier BioMarin to work together to find a solution to provide the drug.
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