DiscoverPatient Empowerment Program: A Rare Disease PodcastSneak Peak of the 2025 Nano-rare Patient Colloquium
Sneak Peak of the 2025 Nano-rare Patient Colloquium

Sneak Peak of the 2025 Nano-rare Patient Colloquium

Update: 2025-09-24
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As the 2025 Nano-rare Patient Colloquium approaches, we welcome back Transmitter Features Editor Brady Huggett to guest-host this episode of the Patient Empowerment Program podcast. Brady sits down with n-Lorem founder and CEO, Dr. Stan Crooke, to reflect onto reflect on the strides made over the past year—insights that will help shape the discussions at this year’s Colloquium.


On This Episode We Discuss:


2:10 How n-Lorem decides to give a medicine specifically designed for one person to another individual


8:10 n-Lorem built systems to be able to treat more than one patient with a single drug


11:08 n-Lorem creates medicines for nano-rare genetically caused forms of ALS


15:14 New suggestions about the plasticity of the central nervous system and the future of medicine


19:40 Why science isn’t in a good place and will experience lasting repercussions


25:00 The Colloquium will focus on what we’ve learned at n-Lorem and what was once thought to be impossible is possible


27:34 An update on the quantity of INDs filed and patients treated with an n-Lorem ASO


28:80 The benefits of implementing whole genome sequencing (WGS) at birth


30:37 What surprises have emerged throughout the organizational journey of n-Lorem


 


NRPC25 registration: https://www.nlorem.org/nano-rare-patient-colloquium-2025/ 


 


Sponsors


ChemGenes: https://www.chemgenes.com/ 


Honegene: https://www.hongene.com/en 

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Sneak Peak of the 2025 Nano-rare Patient Colloquium

Sneak Peak of the 2025 Nano-rare Patient Colloquium

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