Patient Empowerment Program: A Rare Disease Podcast

n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)

Sneak Peek of the 2026 Nano-rare Patient Colloquium

10 EYL, 202647 DAK

Bölüm Hakkında

A lot can happen in a year, and at n-Lorem, this past year has brought important milestones, new developments, and plenty to discuss at the upcoming 2026 Nano-rare Patient Colloquium.  In this special Colloquium preview episode, Brady Huggett, editor-in-chief of Asimov Press and longtime moderator of the Colloquium’s patient experience panel, returns to sit down with n-Lorem CEO Stan Crooke. Together, they reflect on the past year, explore some of the topics likely to take center stage, and preview what attendees can expect at the 2026 Nano-rare Patient Colloquium.  On this episode: 2:28 – What is a day in the life of an n-Lorem research team member?  9:08 – How many ASOs are required to move into tolerability studies and what may cause a program to be terminated  12:05 – Improvements in efficiencies, bringing down costs, and growth have allowed n-Lorem to respond to the extraordinary demand  13:14 – Discussing the creation of individualized ASOs for two boys with SCN2A mutations and their potential to help others with the same mutation and single nucleotide variant  25:00 – Thoughts on the FDA’s Plausible Mechanism Framework  29:00 – Commercial opportunities will not alter the n-Lorem charitable arm but will provide sustainable revenues to charitably treat more patients  36:05 – What are the Limits of Hope and expanding those limits  37:54 – Empathy is an expanded sense of self, and a sphere of oneness is felt at the Colloquium    Links: 2026 Nano-rare Patient Colloquium  Donate to n-Lorem   Episode and NRPC Gold Sponsors: ChemGenes GondolaBio Hongene Biotech