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Treatment for children with spinal muscular atrophy recommended by NICE

NICE has recommended a treatment for children with the rare genetic disorder spinal muscular atrophy (SMA). They can now be treated with nusinersen following the agreement of a deal between the company, Biogen, and NHS England.

NICE has recommended a treatment for children with the rare genetic disorder spinal muscular atrophy (SMA). They can now be treated with nusinersen following the agreement of a deal between the company, Biogen, and NHS England. SMA affects the nerves in the spinal cord controlling movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing. People with the most severe forms of SMA usually die before the age of two years. There are currently no active treatments targeting the underlying cause of SMA so the condition is managed through supportive care that aims to minimise the impact

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