TreatSMA were fortunate enough to be invited to the event along with having a stand for people to come and talk to us about our journey fighting for treatments for rare diseases.
The event was held at the world-famous Wellcome genome campus in Cambridge and had a huge number of speakers from industry, scientific and patient arenas. Talks arranged from looking back at 25 years of rare disease treatment development, talking about recent successes (SMA was mentioned), as well as how the process can change going forward. There were lots of panel discussions around patient involvement and how technology has a huge role to play in the future.
Baroness Nicola Blackwood also attended the event and gave a short talk focusing on what matters most to patients and also looking at what priorities should be set in the upcoming 2020 strategy framework for UK Rare Disease.
It was an enlightening event and hopefully provided research organisations and Baroness Blackwood food for thought.