N=1 Collaborative

Today, individualized medicines are possible.

We are on a mission to make them safe and rapidly accessible to patients worldwide.

Upcoming Seminars

PRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting

Monday, April 22 2024 12:30 pm US EDT Early onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is caused by...

Our Story

What started as a race against time for one little girl has turned into a movement that could help solve the global health crisis of rare genetic diseases.

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Our Work

We are advancing critical steps in the development of individualized medicines, keeping the sharing of knowledge, data, safety and experience at the core.

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Who We Are

We are a group of internationally acclaimed doctors, researchers, patients and companies from across the globe who are committed to expanding the field of individualized medicine.

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Resources & Articles

Publications

A framework for individualized splice-switching oligonucleotide therapy

Jinkuk Kim, Sijae Woo, Claudio M. de Gusmao, Boxun Zhao, Diana H. Chin, Renata L. DiDonato, Minh A. Nguyen, Tojo Nakayama, Chunguang...

Articles & Podcasts

A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments

Bree Iskandar (STAT) July 12, 2023 When Timothy Yu developed milasen, a custom drug for a young girl named Mila with Batten disease, he...

Articles & Podcasts

They Created a Drug for Susannah. What About Millions of Other Patients?

By Erika Check Hayden (New York Times) Photographs by Brittainy Newman Scientists have made rapid progress in customizing drugs for...

N=1 Collaborative

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