Treatment
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
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
