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<a class="text-decoration-none" href="/posts/8010954-advocating-for-people-with-gastrointestinal-stromal-tumor"><div class="Episode-list-grid card-body pan">
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Advocating for People With Gastrointestinal Stromal Tumor
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Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Peter Knox, senior research director at the Life Raft Group. Based in Wayne, New Jersey, this organization was incorporated in 2002 and advocates for people with all forms of gastrointestinal stromal tumor, or GIST.
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31m
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<span><span class="js-time" data-epochtime="1630425420" data-format="short_date">Aug 31, 2021</span></span>
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31m
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<a class="text-decoration-none" href="/posts/8010955-dr-mark-brantly-on-treating-aatd-and-the-work-of-the-alpha-1-foundation"><div class="Episode-list-grid card-body pan">
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<img srcset="https://audioboom.com/i/38818050/75x75/c 75w, https://audioboom.com/i/38818050/150x150/c 150w, https://audioboom.com/i/38818050/300x300/c 300w, https://audioboom.com/i/38818050/600x600/c 600w, https://audioboom.com/i/38818050/1400x1400/c 1400w" sizes="(max-width: 400px) 64px, 96px" style="background-color: #ddb4b1; max-width: 96px; width: 15cqw;" class="js-img-bg ratio-square rounded-1" loading="lazy" src="https://audioboom.com/i/38818050/1400x1400/c" />
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2: Dr. Mark Brantly on Treating AATD and the Work of the Alpha-1 Foundation
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Dr. Brantly is the vice-chair of research in the Department of Medicine at the University of Florida in Gainesville, and has spent most of his career researching a cure for alpha-1 antitrypsin deficiency (AATD).
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12m
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<span><span class="js-time" data-epochtime="1629226811" data-format="short_date">Aug 17, 2021</span></span>
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12m
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<a class="text-decoration-none" href="/posts/8010956-ultragenyx-cmo-dr-camille-bedrosian-discusses-the-first-fda-approved-treatment-for-lcfaod"><div class="Episode-list-grid card-body pan">
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<img srcset="https://audioboom.com/i/38818050/75x75/c 75w, https://audioboom.com/i/38818050/150x150/c 150w, https://audioboom.com/i/38818050/300x300/c 300w, https://audioboom.com/i/38818050/600x600/c 600w, https://audioboom.com/i/38818050/1400x1400/c 1400w" sizes="(max-width: 400px) 64px, 96px" style="background-color: #ddb4b1; max-width: 96px; width: 15cqw;" class="js-img-bg ratio-square rounded-1" loading="lazy" src="https://audioboom.com/i/38818050/1400x1400/c" />
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1: Ultragenyx CMO Dr. Camille Bedrosian Discusses the First FDA-Approved Treatment for LCFAOD
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Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Camille Bedrosian, MD, chief medical officer and executive vice president of Ultragenyx Pharmaceutical in Novato, California. Earlier this year, Ulragenyx won approval from the US Food and Drug Administration (FDA) for triheptanoin (Dojolvi®), a liquid medication that supplies medium-chain, odd-carbon fatty acids as an ener...
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14m
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<span><span class="js-time" data-epochtime="1628691149" data-format="short_date">Aug 11, 2021</span></span>
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14m
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