top of page

Search

N1C VARIANT Guidelines and Tools

Variant Assessments Towards Eligibility for Antisense Oligonucleotide Treatment

Please reference preprint for publications

Access Preprint

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

Eligibility calculator

Video examples

A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases

Olivia Kim-McManus, Joseph G. Gleeson, Laurence Mignon, Amena Smith Fine, Winston Yan, Nicole Nolen, Scott Demarest, Elizabeth...

Phage Therapy for Multidrug-Resistant Bacterial Infections: Past and Future

SNP-selective siRNA for the treatment of Fibrodysplasia Ossificans Progressiva

Comments

bottom of page