A drug keeping two Newcastle sisters alive will be made available on the NHS after the health service struck a deal with manufacturers.
Gail and Matthew Rich have spent more than two years battling for their daughters - and other UK children with the same disease - to have access to a potentially lifesaving treatment on the health service.
Now their campaign has come to the triumphant end they wanted after the National Institute for Health and Care Excellence (NICE) ruled that a specialist treatment known as cerliponase alfa will be available for children with their daughters' condition.
Confirmation of the deal to bring the treatment onto the NHS follows negotiation between NHS England and the manufacturer Biomarin who have agreed a fair price that the NICE committee was able to recommend as an effective use of NHS resources.
The announcement from NICE and NHS England brings to an end two years of devastating setbacks and frustrating delays - but the Rich family say they never gave up hope.
When Nicole and Jessica Rich, from Throckley, Newcastle , were diagnosed with Batten's disease within months of each other, their parents believed it was effectively a death sentence .
But they refused to give up on their little girls and the past few years have seen Nicole and Jessica defy the odds to make medical history.
Since January 2017, Nicole, now seven, has been receiving pioneering treatment at London’s Great Ormond Street Hospital (GOSH) after clinching a place on a trial.
And several months later Jessica, now three, became the youngest person in the world to receive the treatment after getting onto a sibling trial in Hamburg, Germany.
Since she started her treatment Nicole has not shown any additional symptoms of Batten's disease, while Jessica has not developed any signs of the condition at all.
Their daughters' remarkable progress meant Gail and Matthew had the evidence they needed to show that the treatment worked and should be available for all children with the disease.
They launched a tireless campaign, refusing to accept a 'no' from Nice on two previous occasions and even launching a legal battle to challenge the latest refusal.
Now their efforts have paid off with NHS England saying a deal has been struck to give children with rare diseases vital, life-changing treatment. The drug to treat Batten Disease will be available to patients this year.
As well as potentially saving and prolonging the lives of other children with Batten's disease, the news means that Nicole and Jessica's future on the treatment is assured and no longer "at the mercy of a pharmaceutical company".
Simon Stevens, NHS Chief Executive, said: “This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments.
"Over recent months there’ve been a series of successful deals as NHS England works closely with the life sciences sector to make life changing new drugs available for haemophilia, MS, rare cancers and other conditions.
"Coming after extended negotiation, the new deal reached today is a reminder that in order to succeed, companies must be flexible and realistic, because the NHS in England cannot and will not simply write blank cheques at taxpayers’ expense."
Gail posted on Facebook: "The day we have been praying for has came!!! We can’t stop crying !!!!"
The girls' strength and resilience has touched hearts around the world since ChronicleLive first told their story in 2016, and now they have received 12 nominations for our Chronicle Champions awards in the Champion Child of Courage category.
"Without a doubt, they've changed the world from a treatment point of view," said Gail.
"They have shown the world what can be achieved.
"Looking at Nicole standing up - children with Nicole's condition at her age wouldn't be doing that if it wasn't for this treatment.
"With Jessica, the treatment had never been done on a child that young so the eyes of the world were on her, and she showed that it can change the course of the disease.
"It's changed what people think of Batten Disease because it's always been fatal, and in the other types of the disease it is always fatal, but Jessica is still showing no symptoms."
On the award nomination, Gail said: "It's lovely news. They absolutely do deserve it because they're dealing with something so horrendous and they're the strongest little girls
"Hospital is all they've ever known, and it's not what you would want for your child, but they're in a routine and they give us the strength, because nothing fazes them.
They've inspired others, they've had so many lovely reactions from the community. In a way they've brought the community together and they've no idea of the influence that they've had.
"When our MP Catherine McKinnell was standing up in Parliament fighting for us she said everyone in Newcastle knows who Nicole and Jessica are, and we get messages on our Facebook page from people who are so touched by the girls."
On the rollercoaster the family have been on over the past three years, Gail said: "It's changed our lives because materialistic things are just not on our radar - all we want is to be together with our children.
"Their story is bringing out the best in people and showing how you can go back to basics with what's important in life."
