The Digest:
A groundbreaking and extraordinarily costly medical treatment has transformed the life of a young boy in England. Five-year-old Edward, who was diagnosed with the severe muscle-wasting condition spinal muscular atrophy (SMA) as a baby, can now walk independently after receiving a one-off £1.79 million gene therapy on the NHS.
Key Points:
- Edward was one of the first children in England to receive Zolgensma, a gene therapy for SMA, through the NHS in 2021.
- SMA typically leaves babies unable to live past two years without intervention, causing profound muscle weakness.
- His mother, Megan, said Edward has gone from being lethargic to a "cheeky, playful boy" who is now starting school.
- He can now walk 20-30 steps independently and has even learned to swim and float despite the condition.
- The family moved to London and fundraised £170,000 to pay for intensive private physiotherapy, which they credit for his progress.
- Megan gave up her career in event management to become Edward's full-time carer.
- NHS England's medical director praised the "remarkable benefits" seen in Edward and over 150 other children treated with Zolgensma.
Sources: BBC News
