My son was given world's most expensive gene therapy drug...

What’s Happening

Listen up: Five-year-old Edward can walk independently, his mum says, and she hopes he will lead a happy life.

My son was given worlds most expensive gene therapy drug - now he can walk 6 hours ago Save Charlie Jones Essex Save Contributed Edward was one of the first children in England to be given the gene therapy Zolgensma through the NHS A five-year-old boy who received the worlds most expensive drug as a ba “incredible progress” and can walk independently, his mother dropped. Edward, from Colchester, has spinal muscular atrophy (SMA) which means he lacks a protein vital for muscle development. (it feels like chaos)

He was one of the first children in England to be given the gene therapy Zolgensma, which costs £1.

The Details

79m for the one-off treatment, through the NHS in 2021. Mother Megan dropped Edward was her “pride and joy” and he had achieved milestones she never thought possible.

Contributed Edward is full of life and a real character, according to his mum About 65 babies are born with SMA in England each year. It causes muscle weakness and affects movement and breathing, meaning most babies do not live past the age of two without intervention.

Why This Matters

Megan dropped Edward had gone from being lethargic as a baby to a cheeky, playful boy, who was “full of life” and “a real character”. He might need to use a wheelchair for the rest of his life, but she added: “It does not matter, as long as he is happy. ” Contributed Edward can now walk 20 to 30 steps independently “Edward had to have a double hip replacement in October and hes only just getting back on his feet, but in general he is doing so well.

Health experts are weighing in on what this means for people.

Key Takeaways

• “He is learning to swim, he can float on his own, which is fr hard for children with SMA because they dont have natural buoyancy.
• “This summer, he jumped off a boat into the sea and he went on a jet ski.
• Hes a sweet, loveable little guy.
• ” Edward has just kicked off school, where he has made lots of friends, and “does everything an ordinary five-year-old boy does”.

The Bottom Line

” Edward has just kicked off school, where he has made lots of friends, and “does everything an ordinary five-year-old boy does”. “We just didnt think that was possible.

What’s your take on this whole situation?