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N1C VARIANT Guidelines and Tools

N1C Moderator
Sep 26, 2024
1 min read

Updated: Mar 31, 2025

Variant Assessments Towards Eligibility for Antisense Oligonucleotide Treatment

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Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments

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Eligibility calculator

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Video examples

Watch Workshop Recording

NIH Support for the development of therapeutic platform approaches to treat monogenic disease

NIH Support for the development of therapeutic platform approaches to treat monogenic disease

Deciphering SPTAN1-associated disease mechanism and potential therapy in patient-derived cell lines

Deciphering SPTAN1-associated disease mechanism and potential therapy in patient-derived cell lines

N=1 Collaborative: FDA’s New Plausible Mechanism Pathway

N=1 Collaborative: FDA’s New Plausible Mechanism Pathway

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