Solid Science Summit

What's your motivation? It's a strange question to ask someone like me. I think it's pretty obvious. But here's another question: does your motivation change? And the answer, I think, is yes. Well it certainly has done for me.

I founded Joining Jack because I wanted to save my son's life. But along the way I happened to fall in love with a community, the Duchenne community. This has changed things for me, and took me in a direction I didn't intend to go.

I have been questioned about some of my advocacy decisions by family, friends, clinicians and researchers. I ended up lobbying for the reimbursement of Translarna, a drug that won't benefit my son, but having followed closely Archie, Ben and Cormac I couldn't help but feel for their plight. And then there is Sarepta's drug eteplirsen. I don't think an exon skipping drug will come in time to benefit my son, but for Sam, Felix, Toby, Seth and many boys from the UK who could benefit, I have made those flights.

I have been in Boston for three days, having left behind my beloved boys on a quest to find a treatment for Jack and everyone else living with Duchenne.

I have spent two days working intensely with Solid Biosciences, attending a research summit they kindly hosted. What struck me was their motivations. I do genuinely find them completely different from other companies. They WANT to put patients completely at the centre of the drug development process.

I think Solid is different because the founder, Ilan, is the dad of a boy with Duchenne. He wants good, safe and effective drugs, because he ultimately wants a treatment that he can give to his son too. A motivation I understand and feel comfortable with.

I am really excited to be working with Solid Bioscience, and would encourage the community to have open dialogue with them. I believe they actually want to hear from the patients. They understand that we are the experts in this disease and want to tap into our knowledge. They see us as equal partners in this process.