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N1C VARIANT Guidelines and Tools

Variant Assessments Towards Eligibility for Antisense Oligonucleotide Treatment

Please reference preprint for publications

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

Eligibility calculator

Video examples

Positive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies

N=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth

UNC Fast-Tracks Personalized Treatment for Twins with Ultra Rare Genetic Disorder

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