In this special Best Of episode, we revisit some of the powerful moments from the 2025 Nano-Rare Patient Colloquium—an extraordinary gathering that brought together more than 875 patients, family members, scientists, physicians, advocates, and industry leaders from around the world, both in person and virtually.
This episode highlights moving patient stories, thought-provoking scientific conversations, and community-led discussions that reflect the momentum building across the nano-rare space. Together, these voices showcase the growing impact of personalized experimental medicines and the shared determination to turn possibility into progress.
From inspiration to action, this Best Of captures the heart of the Colloquium—and the collective commitment to creating a brighter future for individuals and families living with nano-rare diseases.
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Watch on YouTube:
On This Episode We Discuss:
- 1:15 Expert Panel – Complexities in Long-Term Evaluation of Pediatric Neurodevelopmental and Neurodegenerative Disease
- 5:35 Patient Panel – In Their Words: Diagnostic Journeys and the n-Lorem Experience
- 15:54 Expert Panel – Navigating the Complexities of Individualized ASO Development
- 21:02 Supporter Panel – Enabling the Mission: How the Community Can Help Advance n-Lorem’s Impact
- 26:33 Community Panel – Why Community Matters: A Conversation with Nano-rare Patients and Families
- 32:40 Physician Panel – Redefining What’s Possible: Clinical Perspectives on Developmental Gains in Treated Patients
- 36:57 Patient Perspectives – Finding the Right Partner in Care
- 42:20 Stan Crooke Closing Remarks
Credits
Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Kira Dineen, Jon Magnuson, Andrew Serrano and Amy Williford.
More from n-Lorem:
See what else we are up to on X, Instagram, Facebook, LinkedIn, YouTube and our website, nlorem.org. If you enjoy this episode, please rate and review us, as it helps others find our podcast. Questions/inquiries can be sent to [email protected].
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We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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