We're all Joining Jacks fight against Duchenne Musculare Dystrophy, are you?

Solid Science Summit

What’s your motivation?
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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. Yes I think it does. 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 direction I didn’t intend to go. A complicated, burdensome journey that leaves me with endless worry and sleepless nights, and gave birth to Duchenne UK, something I am incredibly proud to have been part of founding. We all, always, want to do the right thing. 

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 now Tilly a little girl with Duchenne who could benefit from this drug, I wanted to help them all get their chance to have the right to try.  And then there is Sarepta’s drug eteplirsen. I am not at the epi centre of this mammoth advocacy effort, but I have made countless trips to the US to advocate for the approval of this drug. And yet again it won’t help my son. I actually don’t think an exon skipping drug will come in time to benefit my son. Jack has a rare mutation. But for Sam, Felix, Toby, Seth and many boys from the UK who could benefit from eteplirsen, I have made those flights, because they have captured my heart and made me support this cause. Advocating for these drugs has also taught me so much. It has been a steep learning curve. I am now a fully paid up member of the right to try cause and on a mission to find a treatment for all of this community. It often feels slow,  but we are making steps forward.   

I am once again blogging on a flight home from the US. I have been in Boston for 3 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 and left me laid awake last night 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 find some companies in the Duchenne space nod their heads and say they understand, and will make changes to their protocols and advocacy efforts, but they never do. And that is making our community suffer. This is something I am growing very tired of, and I am often left wondering how we put a stop to this.  

I think Solid is different because the founder, Ilan. He 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. 

For the first time during this trip I got to interact with Solid’s board. If I am perfectly honest I don’t think they're your usual investors in Duchenne drug development (but time will tell). They’ve come into this because they want to change the outcomes of this disease for these patients. I don’t personally believe you can ever put a monetary value on being able to change lives, and I don’t think they are thinking primarily about the money. I think their satisfaction will come when they find an effective treatment for the community and in the process they are willingly making every resource available to make this happen and will only employ people who share the same values.

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 to make sure we develop drugs and devices that are meaningful to us. They see us as equal partners in this process. So my message is this, share your ideas with them, talk to them, they want to listen. 

 

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