New chance at life for Leeds' toddler Connie as funding agreed for Batten Disease 'wonderdrug'

Caroline Day with her daughter Connie Annakin who has a fatal disease called batten syndrome, but will now be able to receive treatment which could delay its onset for decades. Picture and videos by Bruce Rollinson.

Family's plea over Batten's drug to keep Yorkshire toddler Connie alive

Her desperate family have been pleading over urgent access to a revolutionary new treatment which could stave off symptoms for decades, but it was deemed too expensive.

Now, days before the decision was to be challenged in the High Court by families nationwide, NHS England has announced that a funding deal has finally been struck.

It will mean that Connie could be gifted an extra 30 years of life, with treatment to begin by Christmas at the latest.

"We can't really take it in," said her emotional mother Caroline Day as she heard the news.

"I just keep thinking of all the things she will now live to do - she will go to school. She will learn to ride a bike.

"These are all the things we never thought would be possible. This is giving her a life back."

Connie Annakin, aged three, who will now be able to access treatment for Batten Disease on the NHS. Picture Bruce Rollinson

Batten disease and the battle over treatment

Batten disease is an incurable and degenerative illness, which causing seizures, loss of sight, mobility loss and then dementia and early death.

While there is no cure, there is a new treatment. In trials, it was found that enzyme replacement therapy, from BioMarin, could halt the effects of the disease, giving children an additional 30 years of life.

But it is expensive, and at a price of £500,000 a year it was deemed not cost-effective by advisory body NICE, and was therefore not recommended as a treatment for the NHS.

Caroline Day with Connie, Connie's brother Elliott, 13, and grandmother Mavis Day. The family, from Farsley, are overjoyed to hear that Connie will now be able to access treatment for Batten Disease on the NHS. Picture Bruce Rollinson

Families nationwide have been fighting this decision and, as discussions reopened between the NHS and BioMarin, two sets of parents were preparing for a High Court battle.

Today, as the news broke that Connie is to be offered the treatment at Great Ormond Street Hospital, Miss Day, 41, wept with joy.

"Thank you so much, for highlighting Connie's fight, that has gone such a long way to making a difference," she said. "I'm absolutely over the moon."

The family are currently on what they feared would be their last ever holiday, in Spain. They had been on a cycle ride when the calls started coming in to say Connie would be offered treatment.

"I'm overwhelmed, we're so elated," said Miss Day. "I can't stop thinking of those children for whom it came too late - there's no way to make that right.

"At least this fight, and today's decision, means no other parent in the future will have to go through what we did."

Today's announcement has been hailed by the NHS, NICE and by Health Secretary Matt Hancock.

Chief executive of NHS England, Simon Stevens, said the announcement was a concrete step towards ensuring patients with rare conditions get access to important new treatments.

"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," he added.