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Hanging Our Hat on Shared Learnings

February 2023

Researchers, clinicians, families and companies came together to create the N1C based on the core principle of sharing learnings to advance the field of individualized medicine as safely and efficiently as possible. Collaboration and sharing were at the heart of the effort that made milasen possible, and that collaborative approach will be necessary to make the individualized medicine approach routine worldwide and bring genetic treatments to the patients that deserve them.

We are building databases to enable this type of data exchange, and we are signing data sharing agreements with institutions and companies around the world. Now, we are announcing the launch of an open letter -- the Data Sharing Pledge for Orphan Therapies -- to the entire rare disease community to affirm support for this critical commitment.

YOU can help!

Data Sharing Pledge for Orphan Therapies

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

Data Sharing Agreement

Sign the Data Sharing Agreement (DSA) to contribute and access data shared among participating institutions for research and educational purposes.

Data Sharing Seminar

Join our Seminar on February 27 (below) to learn more about how together, we can learn and improve from each other for the benefit of the patients who need us most.

Upcoming Seminars

Sign up now for our seminars coming up this month hosted on Mondays, 10:30-11:30am US ET:

  • February 27 - Data Sharing & an Open Letter” by Scott Demarest MD MSCS (Clinical Dir. Precision Medicine, Children's Hospital Colorado), Jonathan Watts (Prof., RNA Therapeutics Inst., UMass), Nina Green (Sen. Adv. to Boston Children’s Hospital Tech Innovation and Dev Office), and Ashley Kuniholm (Regulatory Affairs Manager, Boston Children’s Hospital)

Last Month’s Seminars

Thank you to those who attended our Seminar Series kick-off last month! We were encouraged by the participation of hundreds of N1C members. Please reach out to Erin McConnell at with any feedback or interest in presenting topics or (de-identified) cases:

  • Jan. 23 - Winston Yan, President of N1C - “An Overview of the N=1 Collaborative” and Tim Yu, Geneticist & Neurologist, Boston Children’s Hospital - “ASO Treatment for KCNT1

  • Jan. 30 - Jeff Milton, CEO of La Jolla Labs - “Models for Sustainable Discovery”

We are continually grateful for your support and thank you for signing the pledge today and encouraging your institution or company to sign the DSA.

- N1C Team


We envision a future where individualized medicine centers around the world routinely offer patients customized treatments targeting their condition’s underlying genetic cause.



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