Addressing the needs of nano-rare patients: the n-Lorem experience

Stanley T Crooke, Sarah Glass, Joseph G Gleeson, Laurence Mignon, Konstantina Skourti-Stathaki, Julie Douville, Megan Knutsen, He Pu, Jennifer M Bain, Elizabeth Berry-Kravis, Neil A Shneider, Olivia Kim-McManus, Florian S Eichler, Wendy K Chung, Amanda Nagy, Horacio Kaufmann, Alejandra Gonzalez-Duar · 2026

Read Addressing the needs of nano-rare patients: the n-Lorem experience by Stanley T Crooke, Sarah Glass, Joseph G Gleeson, Laurence Mignon, Konstantina Skourti-Stathaki, Julie Douville, Megan Knutsen, He Pu, Jennifer M Bain, Elizabeth Berry-Kravis, Neil A Shneider, Olivia Kim-McManus, Florian S Eichler, Wendy K Chung, Amanda Nagy, Horacio Kaufmann, Alejandra Gonzalez-Duar (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13227102.txt.

Description

Some patients are one in thirty million, and a 2026 paper by Stanley Crooke and co-authors describes a nonprofit built to treat them one at a time. The group, n-Lorem, designs custom antisense drugs for people whose mutation is known in fewer than thirty patients worldwide, and gives them free for life.

The paper lays out how the foundation finds patients, judges whether the drug approach fits, and makes each bespoke medicine. It profiles the first 329 applicants whose reviews were complete and sums up treatment results so far. Fuller safety data on each drug is promised in companion papers.

Catalog record

CREATORStanley T Crooke, Sarah Glass, Joseph G Gleeson, Laurence Mignon, Konstantina Skourti-Stathaki, Julie Douville, Megan Knutsen, He Pu, Jennifer M Bain, Elizabeth Berry-Kravis, Neil A Shneider, Olivia Kim-McManus, Florian S Eichler, Wendy K Chung, Amanda Nagy, Horacio Kaufmann, Alejandra Gonzalez-Duar
YEAR2026
COLLECTIONPAPERS
FORMATTXT
FILE SIZE89 KB
VERSION1.0
LICENSECC BY 4.0
RIGHTSCC BY
SOURCEEurope PMC

Source: https://europepmc.org/articles/PMC13227102

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