N=1 Collaborative

Today, individualized medicines are possible.

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

Data Sharing Pledge for Orphan Therapies

Sign our pledge and support the fundamental step of transparently sharing data in rare disease:

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Upcoming Seminars

May 3, 2023 2:00-3:00 pm US EST

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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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News & Updates

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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