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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