Take a glimpse behind the curtain as we let you in on a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re excited to welcome back longtime biotech journalist and now features editor of The Transmitter, Brady Huggett, to the podcast for interview of n-Lorem CEO, Stan Crooke, previewing the upcoming Colloquium. Together, they’ll highlight key topics and emerging trends that will shape the conversation at the Colloquium and around nano-rare diseases in the year ahead.

• 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 treatment 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

Brady Huggett is the features editor at The Transmitter, and also hosts the podcast Synaptic. Previously, he served as business editor at Nature Biotechnology. Brady has a B.S. in biology from Wake Forest University in Winston-Salem, North Carolina, and an M.A. in journalism from the University of North Carolina at Chapel Hill.

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