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FARAFARA Cure FA

Roundtable for Rare Disease

ARMroundtableAs Rare Disease Day 2016 is today (February 29), it is only fitting that I talk about my experience speaking  at the Alliance for Regenerative Medicine’s (ARM) Roundtable for Rare Disease (alliancerm.org/event/arms-rare-disease-roundtable) at the end of January. This event was cohosted by FARA. Along with doing much of the emceeing, FARA’s (our) Ron Bartek also chaired a panel discussion on collaborating on cures for rare disease.

When Jen Farmer asked me to speak, I was incredibly honored and immediately said yes. Then, I realized I had to say something and in order to say it well, I should know what regenerative medicine is and what ARM does. As the name regenerative indicates, regenerative medicine is focused on making organs and tissue that have been damaged by disease, injury, and aging well again. (Three cheers for that I say!!) This may sound like medicine as we know it, but what makes this approach unique is that it is inclusive of not just biological science, but also technological and chemical science. So, it doesn’t let medicine lay claim to improving the lives of patients, it says we need lots of collaborators to do that and we shouldn’t just be looking to one field. Collaborators. That’s what ARM does (FARA too!), advance regenerative medicine by bringing potential and active collaborators together. And, at its essence, to me, that is what the roundtable was about. How do patient advocates, patient groups, academics, and pharmaceutical companies collaborate to cure disease? 

So I had a good idea of what the event was about and set to preparing to talk. I knew a little of my story was essential. I also wanted to talk about the difference between knowing a cure is important and feeling it is important. I think feeling it is much more motivating and that is best done by knowing patients. I also wanted to help non-patients see that patients can be helped right now by being part of the process of finding a cure. We are empowered by such experiences and empowered patients are healthier patients. Kyle Bryant helped me fine tune what to say.

I decided to stretch myself by traveling to DC from MA, including an overnight stay, on my own. I was nervous, it has been quite a while (like pre-wheelchair) since I did that. I decided that asking for help when I needed it was the key and it was. Happily, I didn’t have any problems – or major scary problems.

The roundtable was great! So many REALLY smart, accomplished, and thoughtful people genuinely seeking ways to meaningfully and appropriately involve patients were there. Some important questions were: How do we find patients? How do we harness the patient voice for use in the drug approval process? What is a meaningful outcome for a patient and how do we learn that? What is/are there appropriate roles for all the collaborators?

On top of being able to learn about regenerative medicine, I was lucky to find myself surrounded by friends and supporters new and old.

JeanWalsh2015JeanWalshsignature

About the Author

The FARA Ambassador Program

The FARA Ambassador Program

The FARA Ambassadors are a united team of patient volunteers living with FA who are committed to supporting FARA in the search for a treatment and cure.
 
Together we seek to know more about FA, and FARA so we can be prepared to represent the community when the opportunity arises; speaking at events, to volunteers, potential donors, scientific groups, pharma partners, media interviews and other awareness and fundraising opportunities. We believe support is key to continued success toward our ultimate goal of treatment and a cure. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community.
 
The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

 

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