Emergency Medicine Cases – Where the Experts Keep You in the Know. For show notes, quizzes, videos and more learning tools please visit emergencymedicinecases.com
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Addressing Genetic Mutation that Leads to Rare Disease of the Immune System with Paula Ragan X4 Pharmaceuticals
Manage episode 270384237 series 99915
Content provided by Karen Jagoda. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Karen Jagoda or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.
Paula Ragan, CEO and President, X4 Pharmaceuticals is developing innovative approaches to treat patients suffering from rare diseases with an initial focus on WHIM syndrome, a rare disease of the immune system that has a basis in a genetic mutation. X4 is also focused on a rare form of lymphoma called Waldenstrom macroglobulinemia, a blood cancer also caused or driven by a gene mutation. The X4 drug mavorixafor is in capsule form and is a lifelong therapy that revs up the immune system by attaching to white blood cells.
#WHIMSyndrome #WHIM #RareGeneticDiseases #mavorixafor #CXCRA #Waldenstrom
1889 episodes
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Manage episode 270384237 series 99915
Content provided by Karen Jagoda. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Karen Jagoda or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.
Paula Ragan, CEO and President, X4 Pharmaceuticals is developing innovative approaches to treat patients suffering from rare diseases with an initial focus on WHIM syndrome, a rare disease of the immune system that has a basis in a genetic mutation. X4 is also focused on a rare form of lymphoma called Waldenstrom macroglobulinemia, a blood cancer also caused or driven by a gene mutation. The X4 drug mavorixafor is in capsule form and is a lifelong therapy that revs up the immune system by attaching to white blood cells.
#WHIMSyndrome #WHIM #RareGeneticDiseases #mavorixafor #CXCRA #Waldenstrom
1889 episodes
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Similar to Empowered Patient Podcast
Emergency Medicine Cases – Where the Experts Keep You in the Know. For show notes, quizzes, videos and more learning tools please visit emergencymedicinecases.com
…
continue reading
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continue reading
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…
continue reading
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…
continue reading
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…
continue reading
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…
continue reading
Help me fill in the blanks of the practice of ED Critical Care. In this podcast, we discuss all things related to the crashing, critically ill patient in the Emergency Department. Find the show notes at emcrit.org.
…
continue reading
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