'3.4 Billion Won Injection' Administered for Free Works a Miracle... Astonishing Change for UK Boy Who Couldn't Walk

Spinal Muscular Atrophy, a Rare Disease
Treated Thanks to Free Public Healthcare

A five-year-old boy from the UK who received the world's most expensive gene therapy has miraculously regained the ability to walk after four years. This boy suffers from spinal muscular atrophy (SMA), a rare genetic disorder.

On December 31 (local time), the BBC reported that Edward Willis-Hall, a boy living in Colchester, UK, has recovered to the point where he can walk unaided and even swim after receiving an injection of the costly therapy.

Edward Willis-Hall, a 5-year-old boy from the UK suffering from spinal muscular atrophy (SMA), was able to walk after receiving the Zolgensma injection, which costs 3.4 billion won per dose. BBC

Edward was diagnosed with SMA when he was about two months old. SMA is a genetic disease in which the anterior horn cells of the spinal cord are damaged, preventing motor signals from reaching the muscles and causing progressive muscle weakness. This condition primarily affects infants and young children, and for those born with type 1 SMA, the average life expectancy is just two years, making it especially fatal. As time passes, muscle degeneration becomes severe, eventually making even breathing difficult. According to the UK National Health Service (NHS), around 60 to 80 babies are born with SMA each year in the UK.

When Edward was about five months old, he received the SMA treatment 'Zolgensma,' developed by Swiss pharmaceutical company Novartis, with support from the NHS. SMA usually occurs when the SMN1 gene does not function properly, and this drug works by introducing a substitute for the SMN1 gene into the body, enabling continuous production of the necessary protein. The treatment is so effective that a single intravenous injection is sufficient, but the problem is that each dose costs as much as 1.79 million pounds (approximately 3.4 billion won). The UK NHS approved this drug for free public healthcare coverage in 2021.

Thanks to NHS support, Edward was able to receive Zolgensma at no cost. After the injection, his condition improved remarkably. In October last year, he successfully underwent surgery on both hips, which had been misaligned due to weakened muscles, and recently, he has been able to walk 20 to 30 steps on his own. He also started swimming and can now float in the water by himself. For infants with SMA, muscle weakness is usually so severe that they lack the natural buoyancy to float. Being able to float unaided is evidence that his muscles have recovered significantly.

Edward's mother said, "As a baby, he could barely move, but now he is a mischievous and energetic five-year-old boy," adding, "He is reaching milestones we never even imagined." The BBC reported, "Edward started elementary school this year and is doing everything a typical five-year-old does, including making many friends."