Edward, a one-year-old boy from Colchester in Essex, suffers from spinal muscular atrophy (SMA). This means that he is deficient in a protein essential for muscle growth.
In August, he was offered the Zolgensma gene therapy, which is a new treatment that costs PS1.79m.
Megan Willis stated that her son has reached milestones she had never imagined.
She said that he can now sit up on his own, roll over and is able to sit down.
She said, “We are so proud to Edward.” He’s doing an amazing job. He exceeds our expectations. We are so lucky.
“All I wanted for him was to be able sit, and then I knew he would have an incredible life.”
“We have been working with him on his walking for several weeks. He now moves his legs in the gaiters. This is a great achievement.
It brought a tearful to my eyes when I saw it for the first time. It was amazing.
Edward could see a physiotherapist up to five times per week when his family moved to London.
Ms. Willis, who tracks Edward’s progress on Instagram, stated that Edward has transformed from being lethargic and become a normal, cheeky, playful baby.
Edward has found his life back thanks to this drug. He now has a new lease on life.
It’s a marathon and not a sprint. Edward will need to catch up, but he is slowly improving,” she stated.
Zolgensma is believed to be the most costly drug in the world. However, NHS England stated that it had obtained a discount on its PS1.79m price.
Each year, around 65 babies in England are born with SMA. SMA causes weakness in muscles and affects movement and breathing. Most babies don’t live past two years without treatment.
Edward was two months old when he was first diagnosed. He was also receiving Spinraza, which is a drug that requires regular injections to the spine throughout life. This is in contrast to Zolgensma’s one-off injection.
It is a relatively new drug so long-term results are unknown. However, Ms Willis stated that she believes this first generation of SMA babies will reach adulthood.
She said that last year’s time was terrible because he had just been diagnosed with cancer and she didn’t know if he would survive.
The family can now look forward to the next year knowing that he is receiving the best care possible.
“Last year we kept wondering ‘what the future holds for us?’ We thought, “What’s the future?” and then we wondered, “Are we going to lose our baby?”
“Now there’s light, there’s a future. I honestly didn’t believe Edward would have one.
“I used to think his time with us was over, but now I don’t believe that.”
