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First ever treatment for children with rare muscle-wasting condition

 

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First ever treatment for children with rare muscle-wasting condition

The NHS will provide a promising new treatment that can prolong the lives of children with a rare genetic condition under a deal with the manufacturer.

Nusinersen, also called Spinraza and made by Biogen, is the first treatment that targets the underlying cause of spinal muscular atrophy (SMA).The condition affects the nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. Where it develops in babies and toddlers, it can significantly reduce life expectancy. Between 600 and 1,200 children and adults are currently living with the condition in England and Wales. More....