Patient Empowerment Program: A Rare Disease Podcast

In this video message, Chairman Stan Crooke reflects on the close of 2025, six years after founding n-Lorem. He shares his gratitude for the community that made it the organization’s most successful year to date and offers perspective on how progress across every area of the foundation is carrying n-Lorem forward into 2026.

Read the 2026 Chairman Letter

Today, we’re launching a NEW SERIES that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today.

Every day, our patients benefit from what can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time.

They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease.

Today’s miracle: Genomics.

Coming next: iPSCs, the RNA World, and Antisense Technology. The breakthroughs that pushed possibility even further.

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The Patient Empowerment Program podcast is hosted by n-Lorem Founder and CEO, Stan Crooke. Dr. Crooke recently joined the advisory board of the CNBC Cures, an initiative bringing together families, doctors, investors and regulators with one goal in mind: helping to raise awareness of, and improve patient outcomes for, the 30 million Americans suffering from rare diseases. You can join the CNBC Cures Newsletter here: https://www.cnbc.com/cnbc-cures-newsletter/

Today's sponsor is ChemGenes: https://www.chemgenes.com/

Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often is Drug safety monitored? When is an existing ASO made available to others? Not capping accepted applications and more. Do you have additional questions?

Email [email protected].

 

On This Episode We Discuss:

5:40 - What is the frequency range of intrathecal and/or LP administrations? Do the treatments not typically cross the blood brain barrier?

7:06 - How do you administer the ASO to animals - is it according to how you plan to administer to the patient?

7:36 - How do you evaluate the potential on-target toxicity if the nonclinical species does not contain the targeted sequence?

9:35 - How often is safety monitored? Is it only monitored quarterly at the Data/Drug Safety Monitoring Board (DSMB) meetings?

11:03 - How long after an ASO is developed for one child, is it offered to other children? Of course, we know that they will have to submit an application. Does the ASO become potentially available for other children after the first child's 2-year trial is over? Or is it potentially available after just a few doses to confirm safety and efficacy?

14:18 - Are there plans to commercialize drugs for the nano-rare?

16:27 - What proportion of institutions elect to use external / centralized institutional review boards (IRBs)?

18:21 - Will there be a cap of accepted applicants from n-Lorem? The demand will only grow and there’s a fear for some families that there’s a chance of n-Lorem capping the number?

19:44 – Once we have our neurologist team working on getting our child accepted, what else do we need to do? What I guess is our next step? Is there anything we should do in the meantime?

20:31 – What should a parent expect once their application has been accepted to n-Lorem?

21:19 – There are widely varying timelines regarding this process. What as a parent should I expect regarding timing?

22:30 – There are companies and academic scientists that claim to provide ASOs for a fee. What do you think about these providers?

23:40 – Insights: What is n-Lorem learning that will be broadly applicable to the scientific community; Dreaming bigger and hoping for more

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. 

 
Recap page: 

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

 

Colloquium YouTube Playlist (alternative to recap page): 

https://youtube.com/playlist?list=PLrDVyc3t26FxvnqoiApY_Qw1_weTAQ4MS&si=EWBvQ0ZdDH-Rq1mJ

A conversation with Natacha Gassenbach, 2025 Hero of n-Lorem and Biogen leader. She shares Biogen’s decision to become a founding donor of n-Lorem, the impact of the Nano-Rare Patient Colloquium. Natacha also explores “the movement for nano-rare” and a shared vision of tackling difficult challenges to drive meaningful change.

 

Holiday Ornament: https://www.nlorem.org/holiday-ornament-fundraiser/

 

On this episode:

1:18 – Natacha introduction

3:00 – Biogen’s path to becoming a founding donor of n-Lorem and giving back to the community

4:57 – Why Natacha and Biogen invested in n-Lorem and convincing new individuals to buy into the mission

7:19 – Biogen has hosted the n-Lorem Nano-rare Patient Colloquium since 2023

9:40 – Biogen and n-Lorem share the connection of tackling difficult tasks to make a difference in the world

15:05 – A movement for nano-rare the possibilities it may bring

17:45 – Taking advantage of nano-rare learnings and implementing them into drug discovery for larger patient populations