patient identification | N=1 Collaborative

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Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting

Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

Illustrated Examples

N1C Patient Identification Workgroup: Compilation of Useful Tools

A Multistakeholder Perspective on Advancing Individualized Therapeutics

Articles & Podcasts

Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient

Illustrated Examples

The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study

A framework for individualized splice-switching oligonucleotide therapy

Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID

Workshops & Presentations

N=1 Collaborative Introduction & Patient Selection Workshop

OTS Rare Disease N-of-1+ Briefing Document, Jan 2020

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