Edward’s mother, Megan Willis, said that since receiving the drug, her child has seen positive mutations in his condition that she never thought possible.
A baby boy in Britain has been given a chance to live again after receiving the most expensive medical drug in the world.
One-year-old Edward, from Colchester, Essex, had spinal muscular atrophy, which means he lacks a protein vital for muscle growth.
The National Health Service in Britain made available Zolgensma, a new gene therapy costing 1.79 million pounds, to give to Edward in August.
His mother, Megan Willis, said her child has since experienced positive mutations in his condition that she never thought possible.
She added that he was now able to roll over and sit without help, and started taking his first steps with the help of those around him.
“We are very proud of Edward,” Willis said. “He is doing well, beyond expectations. We are very fortunate.”
Edward has received his new gene therapy, which costs 1.79 million pounds
She continued, “The most I wished was that he could sit down, and I knew then that he would have a wonderful life.”
She added, “We focused with him for a few weeks on walking, and he started moving his legs himself, relying on specially designed shoes, which is a wonderful achievement.”
About her feelings, Willis said: “I had tears in my eyes as I witnessed it for the first time…It was a wonderful moment.”
The family had to move to London so that Edward could attend physiotherapy sessions up to five times a week.
Megan Willis and her husband John Hall say they are very happy for Edward’s future
Willis, who documents his treatment journey on Instagram, says that his level of kinetic energy has greatly improved, and he has become a normal, dynamic, playful child after he was inactive.
“This drug brought Edward back to life, it gave him a chance to live again,” she said.
“It’s a marathon, not a sprint,” she said. “It will take a long time for Edward to become normal, and he will reach his goal, but slowly.”
Zolgensma is believed to be the world’s most expensive drug, although Britain’s National Health Service said it had negotiated an undisclosed price reduction.
Every year, about 65 babies are born in England with the problem of spinal muscular atrophy, which causes weakness in the muscles and affects the child’s ability to move and breathe, which means that most of these children cannot live for more than two years without intervention.
Edward, who was diagnosed with the disease at the age of two months, was also receiving another drug called “Spinraza”, which is a regular injection into the spinal cord for life, in contrast to the one-time drug Zolgensma.
The parents had to move to London so that Edward could attend physiotherapy sessions five times a week.
Because it is a new drug, the results of its long-term effectiveness are still unknown. But Edward’s mother says she believes this generation of babies with this health problem will be the first to reach adulthood.
Edward’s family, who lost hope last year in his recovery and life, can look forward to the new year now knowing that their son is receiving the best possible care.
“Last year we’ve been thinking about what the future holds for us, and we’ve been wondering if we’re going to lose it,” Willis said.
