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Drug treatments proven to be effective for SMA

Nusinersen (Spinraza)

Nusinersen was developed by Ionis pharamaceutical company. On 23rd December 2016, it was licensed and marketed in the USA as Spinraza by Biogen pharmaceutical company.

Q and As about nusinersen - is it appropriate for my child?

Q and As about nusinersen's availability in the UK

Where UK Access Has Got To

the UK Expanded Access Programme for children with SMA Type 1

What we are doing to progress access to treatment in the UK

what you can do to help

More detail on how nusinersen works in SMA

key clinical trial results

Spinraza in the United States

What Happens After a Successful Clinical Trial

Dr. Alex Murphy describes the processes that have to be gone through before a drug can be prescribed for an individual in the UK. 


Find out what stage other drugs have reached in our: