• Sneak Peek of the 2024 Nano-rare Patient Colloquium

  • Oct 2 2024
  • Duração: 42 minutos
  • Podcast

Sneak Peek of the 2024 Nano-rare Patient Colloquium

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  • Sumário

  • 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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