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Sneak Peek of the 2024 Nano-rare Patient Colloquium
Manage episode 443177673 series 3349924
Indhold leveret af n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen), N-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen). Alt podcastindhold inklusive episoder, grafik og podcastbeskrivelser uploades og leveres direkte af n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen), N-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen) eller deres podcastplatformspartner. Hvis du mener, at nogen bruger dit ophavsretligt beskyttede værk uden din tilladelse, kan du følge processen beskrevet her https://da.player.fm/legal.
Take a glimpse behind the curtain as we offer you a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re thrilled to welcome back longtime biotech journalist and current Features Editor of The Transmitter, Brady Huggett, to the podcast. In an interview with n-Lorem founder and CEO Stan Crooke, they preview the upcoming Colloquium, diving into key topics that will shape the conversation at the event and around nano-rare diseases in the year ahead.
On This Episode We Discuss:
2:21 How many Investigational New Drugs (INDs) has n-Lorem filed?
5:22 How many patients treated with an n-Lorem ASO have been on therapy long enough to observe a benefit?
6:50 What does it mean for a patient like Susannah who has experienced a significant decrease in behavioral arrest incidents since receiving treatment
9:39 Providing ASOs to patients earlier
13:25 INDs for the ‘n-of-few'
23:15 Introducing Whole Genome Sequencing (WGS) for all newborns
29:05 Stan’s perspective on leadership
33:50 n-Lorem's goals for the 2024 Nano-rare Patient Colloquium
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