Muscular dystrophy toddlers are given hope with new wonder drug Spinraza

Thousands of children paralysed by a cruel muscle-wasting illness are set to benefit from a ground-breaking new drug – the first treatment approved in the UK to save those born with spinal muscular atrophy, a type of muscular dystrophy.

Spinraza, which is injected into the spine, has already helped some sick youngsters walk, talk and breathe easily again.

Others given little over a year to live after being born with the condition are still alive and well since having the drug, long after their second birthdays.

Last week, NHS England announced that the drug would be available to treat the genetic disease that affects an estimated 1,300 British children and adults.

Thousands of children paralysed by a cruel muscle-wasting illness are set to benefit from a ground-breaking new drug – the first treatment approved in the UK to save those born with spinal muscular atrophy, a type of muscular dystrophy. Two-year-old Hune Gascoigne (above), from Stoke-on-Trent, was one of the first patients in England to benefit from the new drug

Thousands of children paralysed by a cruel muscle-wasting illness are set to benefit from a ground-breaking new drug – the first treatment approved in the UK to save those born with spinal muscular atrophy, a type of muscular dystrophy. Two-year-old Hune Gascoigne (above), from Stoke-on-Trent, was one of the first patients in England to benefit from the new drug

The wonder drug, Spinraza, is injected into the spine, and it has already helped some sick youngsters walk, talk and breathe easily again. Above, Hune with her mother, Zeedee Sawyer-Hartley, 27, and Zeedee's partner Ben Gascoigne, 30

The wonder drug, Spinraza, is injected into the spine, and it has already helped some sick youngsters walk, talk and breathe easily again. Above, Hune with her mother, Zeedee Sawyer-Hartley, 27, and Zeedee's partner Ben Gascoigne, 30

The condition, which robs the body’s muscles of their power, varies in severity. The worst and most common form, called type 1, usually leads to death within the first two to four years of life. Less severe types can still cause lifelong suffering, with few able to walk unaided.

The NHS ruling brings England, Wales and Northern Ireland into line with dozens of other countries – including Scotland – where Spinraza has been available for over a year.

‘This is fantastic news,’ said Catherine Woodhead, chief executive of the charity Muscular Dystrophy UK. ‘Children already receiving treatment are reaching milestones never thought possible and living longer. Now hundreds of others will be given that same chance.’

The breakthrough drug could also pave the way for revolutionary new treatments for other muscle-wasting conditions that affect up to 70,000 people in the UK.

The condition, which robs the body’s muscles of their power, varies in severity. The worst and most common form, called type 1, usually leads to death within the first two to four years of life. Less severe types can still cause lifelong suffering, with few able to walk unaided. Hune was diagnosed with spinal muscular atrophy at just six months old when her parents noticed she was ‘floppy’ and losing the ability to grip things

The condition, which robs the body’s muscles of their power, varies in severity. The worst and most common form, called type 1, usually leads to death within the first two to four years of life. Less severe types can still cause lifelong suffering, with few able to walk unaided. Hune was diagnosed with spinal muscular atrophy at just six months old when her parents noticed she was ‘floppy’ and losing the ability to grip things

In a healthy person, a gene called SMN1 produces a protein which forms a protective outer layer around the nerves in the spine that

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