Meet Grace Hoyt — a bright, witty high schooler with a passion for singing, art, and romantasy audiobooks. Grace also happens to live with a rare disease (FLVCR1) that has left her legally blind, unable to feel pain, and unsteady on her feet.

On her 13th birthday, Grace received a first-of-its-kind ASO medicine designed by n-Lorem to preserve her remaining vision. Today, she can still read snippets of her beloved “book children” — the physical versions of her favorite audiobooks — and, importantly, recognize and assess her sometimes life-threatening skin infections, a skill that could save her life.

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

The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem and our nano-rare patients. This honor recognizes our ‘unsung heroes’, those who have gone the extra mile, taken time away from their schedules and families and leveraged their expertise and contacts to support n-Lorem’s mission to provide hope and potential help to nano-rare patients. On this episode of the Patient Empowerment Program podcast, we welcome three of our five 2025 Heroes for a conversation about their inspiration to take action:

- Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics

- Richard S. Geary, Ph.D., Chief Development Officer, Executive Vice President of Development, Ionis Pharmaceuticals

- Scott Henry, Ph.D., DABT, SVP, Nonclinical Development, Ionis Pharmaceuticals

Sponsors:

ChemGenes

Hongene Biotech

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