Improving the UK scene in rare and genetic diseases is no minor task. For example, our country holds the sad record of having the lowest availability of orphan drugs in Western Europe.*
Genetic Alliance works to change this. They try to address many issues: reproductive techniques, diagnosis, medical research, healthcare and delivery, access to treatments, and living and education. You can read about their work here.
They also run the National Campaign for Rare Diseases.
On Rare Disease Day, TreatSMA was approved as a member of Genetic Alliance.
We will now work together to change the country for better for all people with SMA.
* See for instance this report
