N=1 Collaborative

Today, individualized medicines are possible.

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

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

Seminar Series

The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development

February 13 - Annemieke Aartsma-Rus, Dutch Center for RNA Therapeutics - “The most important aspects of the guidelines for N=1 exon...

Seminar Series

Models for Sustainable Discovery

January, 30 - Jeff Milton, CEO of La Jolla Labs - “A Model for Sustainable Discovery in Rare Disease” Millions of patients world-wide...

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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