There are over 300 million people worldwide living with a rare disease. Together across borders, and across the 6000+ rare diseases we work towards more equitable access to diagnosis, treatment, care and social opportunity. The key message for Rare Disease Day 2022 is Share Your Colours.

The team at Rare Disease Day have some amazing plans in place to mark this very special date in our calendar, please keep an eye on social media channels to find out more about how YOU can get involved.

We are delighted to be working in collaboration with Muscular Dystrophy UK to offer our families a unique and exciting opportunity to join us for an informal meet and greet session with some of the PTC Therapeutics team.

What to expect

12.30 – 12.35 – Welcome from Florence Boulton, National Director, Action Duchenne

12.35 – 12.45 – Rob Burley, Director of Care, Communications and Support at Muscular Dystrophy UK will be explaining the process of Duchenne muscular dystrophy (nonsense mutation) – ataluren review of Highly Specialised Technologies (HST) 3 appraisal

12.45 – 13.00 – Hear from Ian Davidson, Regional Head, UK & Ireland at PTC Therapeutics.

13.00 – 13.15 – Hear from Mark Rance, Senior Director of Medical Affairs at PTC Therapeutics

13.15 – 13.30 – Questions

All are welcome and we look forward to seeing you online!