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People with rare bone disease to benefit from life-changing drug

 

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People with rare bone disease to benefit from life-changing drug

Children and adults with a rare inherited bone disease will be able to access a potentially life-enhancing drug, thanks to an innovative deal between NHS England, NICE and manufacturer Alexion.

A new managed access agreement between NHS England and manufacturer Alexion, alongside new draft NICE guidance published this week, will broaden access of asfotase alfa to infants, children and adult patients with paediatric-onset HPP, who experience the most disabling symptoms and are expected to benefit most from therapy.