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

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.