Spinraza NOT approved in England and Wales – SMA patients bitterly disappointed
What a catastrophic decision for more than a thousand people affected by spinal muscular atrophy in England and Wales.
The National Institute for Health and Care (NICE) decided NOT to recommend the use of Nusinersen (Spinraza®) as an NHS treatment for spinal muscular atrophy in England and Wales.
After Denmark, England and Wales is the next country where his patients are locked out of this therapy
We can only guess that the decision related to the recent safety warning on the side effect hydrocephalus
SPINRAZA (nusinersen) EU label will include information on the potential risk of hydrocephalus