Posted by Michael Wonder on 15 May 2019
NICE recommends first ever treatment for children with rare muscle wasting condition
15 May 2019 - Children with the rare genetic disorder spinal muscular atrophy can now be treated with nusinersen after NICE today recommended it following agreement of a deal between the company, Biogen, and NHS England.
Nusinersen (also called Spinraza) is the first treatment that targets the underlying cause of spinal muscular atrophy (SMA).
SMA affects the nerves in the spinal cord controlling movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
