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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

Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder

Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder

RNA/DNA based analytics to guarantee product safety – challenges for N-of-1-treatments

RNA/DNA based analytics to guarantee product safety – challenges for N-of-1-treatments

Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease

Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease

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