Rare diseases

Find out what makes a rare disease rare and who is affected.

Bringing patient support to a rare disease

Learn how we are breaking down barriers that prevent patients from joining clinical trials for spinal muscular atrophy.

Just the other day, I realised I could open the fridge again

Conversations with people with SMA and their families

What patients in clinical trials should know about their data

Patients can be at risk of sharing too much, especially in Rare diseases. How can patient advocates support innovation but also defend patient privacy?

Find out how we determine whether new drugs, diagnostics or procedures in the laboratory are both safe and effective when used to diagnose or treat people.