Treating Spinal Muscular Atrophy

 Not long ago spinal muscular atrophy was a disease with not only no cure but no  treatment,
But all that has changed........

Available Treatments


We used to say No treatment, No Cure but all that changed in 2016 (for the USA) and  30 May 2017 ( for European Union), there is now an effective treatment for Spinal Muscular Atrophy: Spinraza®.  There are also various other  treatments that are in various stages of development.
Gene therapy is also in use in the USA

Spinraza®

Spinraza is the first and only approved medicine for spinal muscular atrophy . It contains nusinersen, it's  main role is to repair the damaged SMN2 gene. 
After treatment with nusinersen, the SMN2 gene starts producing high amounts of  SMN protein, the lack of which is the primary cause of SMA symptoms.
Following a lengthy fight  led By TreatSMA,  in July 2019 Spinraza was approved for all suffers of SMA in the UK by NICE (The National Institute for Health and Care Excellence) the agency responsible for appraising all treatments in the country.

The Annabelle Rose foundation offered assistance and support but we wholeheartedly believe without the work of a team of amazing people this would not have happened. 

There were ups and downs, and a battle with NICE on the particulars of the Managed Access agreement, but the SMA community led by the wonderful Team at TreatSMA remained steadfast and united to make Spinraza available for ALL!!
To find out more about Spinraza and other treatments for Spinal Muscular Atrophy head over to TreatSMA by Clicking below