Parents campaign to extend life of Braunston toddler with rare genetic condition - The Rugby Observer

Parents campaign to extend life of Braunston toddler with rare genetic condition

Andy Morris 22nd Feb, 2018   0

A CAMPAIGN has been launched to help a Braunston toddler with a rare genetic condition whose short life expectancy could be extended – but only by one of the most expensive drugs in history.

Avery Bazin-Ball was diagnosed with type 2 Spinal Muscular Atrophy (SMA) – which causes progressive muscular weakness and loss of movement due to muscle wasting – in November when he was just 18 months old.

Avery, who lives with Mum Lisa, dad Alex, brother Louis and dog Fergie, struggles with breathing and swallowing because of his condition. Without treatment, he won’t ever walk, is likely to lose the ability to sit independently, is at risk of severe complications from chest infections, and his life expectancy is likely to be in the late teens.

Avery (right) lives in Braunston with dad Alex, mum Lisa and brother Louis. (s)

A drug called Spinraza has stopped the disorder progressing and even reversed some of the damage for SMA patients in a number of countries – but it is at least two years away from being approved for use in the UK.

And the drug, developed by pharmaceutical company Biogen, is one of the most expensive in history, costing nearly £540,000 for the initial dose and then £270,000 per year for life.

Alex and Lisa have launched a campaign to raise money for equipment which will let Avery have the best possible quality of life – and to get Spinraza approved in the UK.

In their Facebook blog A Very Special Journey, they said: “It’s difficult to articulate how this situation has affected us as parents – to call it a rollercoaster of emotions seems a dramatic understatement.”

With signs of muscular atrophy already apparent, Avery needs extra support in feeding and playtime, and he is unable to reposition himself when trying to sleep.

“Leading specialists in the UK have confirmed Avery will get treatment in the next few years,” said his parents. “We know we must do everything humanly possible to make sure we keep him strong until this day arrives.

“Treatments already exist that could transform SMA patients’ lives. Surely it’s their right as human beings to have access to this now? They shouldn’t suffer because they were born in the UK rather than America, Germany or South Korea. This injustice is very hard to live with.

“Amazing professionals in NHS England want to do everything they can, but their hands are tied by lack of political will and poor policy making and administration.

“There is no reason why Avery couldn’t live a much longer life than we had initially been told.”

Spinraza has improved rolling and sitting by as much as 40 per cent in children with the most severe type 1 SMA, and last year the European Medicines Agency recommended it should be marketed in the EU for all SMA types.

Biogen has offered it free to type 1 children in the UK and has proposed a short-term agreement which would mean all SMA patients could get it free, with the NHS picking up only the costs of the lumbar puncture procedure to administer the drug.

But there is not yet an agreement to consider Spinraza for NHS England – and the process for approving rare disease treatment would take at least two years.

Visit to make a donation. Visit to follow the campaign and blog, featuring details of events and fundraising activities.

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