Join Stan Crooke, n-Lorem founder, CEO, and host of the Patient Empowerment Program Podcast, alongside Amy Williford, Sr. Director of Communications and Donor Relations, for a special episode.

In this post-colloquium Q&A, Stan recaps the 2024 Nano-rare Patient Colloquium and the progress n-Lorem has made as the organization nears its 5-year anniversary, shares his candid reflections on the event, and dives into additional thoughts and questions that weren’t covered.

Do you have additional questions? Email podcast@nlorem.org.

Question Bank:

1. 2:15 How do you think the event went this year?
2. 6:05 Were observations of benefit expected in patients so quickly?
3. 9:47 What do you expect the number of patients treated to be next year?
4. 10:57 Do you have a message to supporters?
5. 12:30 Were there any disappointments from the colloquium?
6. 13:21 What are the most important things that n-Lorem has learned this year?
7. 16:13 Do you think n-Lorem’s processes will be outlined every year at the event?
8. 18:05 How is AI incorporated at n-Lorem?
9. 19:10 Does your recently announced deal with GondolaBio reflect the sustainability strategy discussed at the event?
10. 20:53 n-Lorem is celebrating 5 years in 2025. Did you expect the foundation to be where it is today?
11. 21:28 Does n-Lorem plan to expand into Europe and elsewhere?

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This year’s Colloquium spanned two perfect autumn days in beautiful Cambridge, Massachusetts where hundreds of nano-rare community members from around the world gathered under a single roof to learn, connect, and support one another. We’re pleased to announce that the event welcomed over 750 attendees, both in-person and virtually. It’s each and every one of you who made the event such a success! For this special episode, we gathered the ‘Best’ clips from the event for you to learn from and enjoy.

To submit questions for our upcoming Q&A episode, email podcast@nlorem.org. We can’t wait to hear from you! 2024 Colloquium Recap

On this episode:

• 2:07 Daphne Graskewicz-Prado, ASO The Perilous Journey to Diagnosis and Treatment for Nano-rare
• 9:35 Ryan Strankowski and Dr, Nelson Leung, Ryan’s Journey and Clinical Experience
• 15:55 Luke Rosen, Megan Hedstrom, and Dr. Jennifer Bain, Susannah’s & Sloane’s Journeys and Unique Clinical Experiences
• 21:49 Kelley Dalby and Dr. Olivia Kim-McManus, Connor’s Journey and Clinical Experience
• 30:00 Dr. Julie Ziobro and Dr. Horacio Kaufmann, Building a Nano-rare Network and Managing Institutional Challenges
• 37:38 Dr. Andrew Lo and Dr. Alan Lotvin, Innovative Ways to Support Nano-rare
• 42:30 Dr. Toby Ferguson, Dr. Liz Berry-Kravis, and Dr. Eugene Shneider, Changing the World One Patient at a Time
• 47:30 Dr. Kate Dawson, What It Means to be a Parter with n-Lorem for Nano-rare
• 50:23 Dr. Stanley Crooke, Providing Hope for a Better Future, One Nano-rare Patient at a Time

Two years of treatment—two years of strength, courage, and blazing a trail for other nano-rare patients! 🎉 Susannah's story was the first shared on the Patient Empowerment Program podcast. Now, two years later, her father, Luke Rosen, and her physician, Dr. Jennifer Bain, detail Susannah’s remarkable progress since beginning regular treatments—highlighting improvements in her motor skills, cognition, energy, and more!

Susannah's Story (May 25, 2022)

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2024 Nano-rare Patient Colloquium

On This Episode We Discuss:

• 3:10 Susannah's KIF1A mutation
• 5:50 Luke and his wife Sally changed their careers to help find a treatment for KIF1A
• 7:23 How Susannah's family first heard of n-Lorem
• 8:43 Jen on the Columbia University Medical Center team and the decision to treat Susannah
• 12:25 Luke's experience bringing his daughter to receive an experimental ASO treatment for the first time
• 14:30 The positive outcomes and observations of Susannah's treatment
• 26:15 What Dr. Bain has learned since treating Susannah
• 29:15 Luke on the impact and importance of the little things
• 31:15 Advice to other parents
• 35:40 Being part of a community of nano-rare patients and families