Epilepsy drug trial 'life-changing for my son'

A mother has said she felt like she had "won the lottery" after a clinical trial transformed the life of her son who has a severe form of epilepsy.

The families of Albie Kelly and Freddie Truelove said the seven-year-olds had been given a new lease of life following the research trial of the drug zorevunersen at Sheffield Children's Hospital.

Both boys have Dravet syndrome - a rare form of epilepsy which often begins before the age of one and affects about one in every 15,000 babies born.

Albie's mother Lauren Kelly said: "Since the research trial it's like I have a new child. It's been life-changing. I feel like a millionaire, like I've won the lottery."

Children taking part in the trial had seen dramatic changes, including taking part in sport, making new friends, and learning to speak, read and write independently, the hospital said.

Albie, who lives with his mother, father and sister in Barnsley, received the first dose of the trial drug zorevunersen in June 2022.

Before the trial, he was non-verbal, showed little emotion and often fell when he walked.

However, after the trial, Albie's family said he had become "incredibly independent" - making new friends at school, playing sport and sleeping through the night.

His mother added: "I used to be terrified to put Albie to bed, never knowing what we'd wake up to. Now, he wakes up and says, 'Morning mummy'".

Freddie, who lives with his mum, dad and younger sister near Huddersfield, was the first child under 13 in the UK to receive zorevunersen in September 2021.

Since then, he has gone from suffering more than a dozen seizures every night, to one or two brief seizures lasting only seconds every three to five days, and even up to 10 days without any seizures.

He can walk up stairs unaided and even swim and ski - something Freddie and his family never thought possible.

His mum, Lauren Truelove, said: "It's really hard to articulate just how big the impact this research trial has had on us, it has completely changed our lives.

"We now have a life we didn't ever think was possible and most importantly it's a life that Freddie can enjoy."

The research trial, based at Sheffield Children's NHS Foundation Trust's dedicated clinical research facility, is led by Stoke Therapeutics.

After an observation period, the children on the trial are given an initial three doses of zorevunersen over a period of 12 weeks.

The second part of the study involves receiving the drug every 16 weeks.

Six patients, from across the north of England and the Midlands, are at Sheffield Children's Hospital on the trial and 17 children are taking part nationally.

Consultant in epilepsy at the trust Dr Archana Desurkar said: "By taking part, children like Freddie and Albie are not just helping themselves, they are helping future generations with these conditions too."

She added that it could take several years before the trial drug became a part of standard NHS treatment due to the rigorous data assessments and regulatory approvals required.

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