Rare Disease Advocates at Work!

Posted on September 29, 2017   |   

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By: Katie Jensen, MPA, Development Manager

This month, I had the chance to go to the Global Genes Rare Patient Advocacy Summit for the first time, thanks to a scholarship designated for rare disease patient advocacy organizations like The LAM Foundation. I spent three days in sunny Southern California connecting, collaborating and learning from other rare disease organizations and the pharmaceutical/biotech companies that specialize in rare diseases.

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I caught up with our friends from the Alpha-1 Foundation, Tuberous Sclerosis Alliance and the Foundation for Sarcoidosis Research, and made some new friends from organizations like the Child Neurology Foundation, Cure SMA and other rare diseases with names almost as complicated as lymphangioleiomyomatosis! Together, we shared how patient organizations can facilitate progress, learned best practices in rare disease nonprofit management and even got some advocating in as we all called our Senators for the Rally for Medical Research.

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We learned about the amazing progress that’s being made in medicine and technology, inspired by speakers like Chris Austin, the director of NCATS. More inspiring though, were the personal stories of progress, like the blind man who helped develop an app that allows him run ultramarathons independently, the friends of a SMA patient who designed a medically-approved backpack to carry him through Europe, and the young woman with cystic fibrosis whose passionate voice inspires other teens across the web. Throughout all of these stories, and the story of The LAM Foundation, the unifying theme was hope. It is clearer than ever that by working together we can find cures, not just for LAM, but for all rare diseases.  

With over 7,000 known rare diseases, it’s easy to be overwhelmed by the amount of work ahead of us. But I’m telling you – this group is going to get it done. The conference was buzzing with talk of CRISPR gene editing, patient registries and strategic collaboration. The Summit also reminded me how small our world is; I had lunch one day with a LAM patient’s accountant, the next day with a gentleman from the CRO that helped us with the MILES trial and ran right into James Valentine, who moderated our recent Patient-Focused Drug Development meeting!

The Global Genes Summit focused on the fact that only 5% of rare diseases have an FDA-approved treatment. LAM is lucky to be part of that group, but as we all know, one approved treatment is not enough. This is why The LAM Foundation continues to fight with you to find better treatments, and ultimately a cure, for this disease.

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All of this progress gives me hope, which I know we will advance at the Patient Benefit Conference & LAMposium LA this November. The Patient Benefit Conference will fund innovative initiatives, like the ones I saw at the Global Genes Summit, to help LAM patients live better lives. We hope you’ll attend, and lend your unique patient voice to help us develop these tools. Join me for my next trip to sunny SoCal by registering today at www.thelamfoundation.org/conference, and bring your sunglasses! The future of LAM is brighter than ever.

To learn more about Katie’s trip, follow her on Twitter @JensenKatieC, or look for #2017GGSummit.

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