Potential Treatments

Drugs Currently Being Tested In Clinical Trials

Drugs Currently Being Tested In Clinical Trials

Track the development of possible SMA treatments in our Drug Pipeline and learn more about some of the drugs currently being tested.

How Clinical Trials Work

How Clinical Trials Work

This information sheet tells you more about how trials work, what to ask, and who to speak to if you are interested in joining one.

The NICE Process - Work to change it and what happens in the rest of the UK

The NICE Process - Work to change it and what happens in the rest of the UK

The appraisal process for nusinersen has clearly been shown to be ‘not fit for purpose’ for rare diseases treatments. Find out what work is being done to challenge this.