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Navigating Institutional Implementation: A Quarterly Open Forum for N-of-1 Clinical Trials
April 10, 2025 at 10:00 am EDT You're invited to this quarterly open forum designed for those with experience or interest in N-of-1...
79 views
Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
David Cheerie , Margaret M. Meserve , Danique Beijer , Charu Kaiwar , Logan Newton , Ana Lisa Taylor Tavares , Aubrie Soucy Verran ,...
52 views
The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK
Daniel J. O’Connor , Parker Moss , Matthew Wood , Martin Murphy , Michael Parker , Nicola Blackwood , Matthew A. Brown , Deb Lancaster ,...
27 views
Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
March 24, 2025 at 12:30 pm US EST We developed personalized splice-modulating ASOs targeting a deep intronic variant in PEX1 in a 2 year...
176 views
RNA/DNA based analytics to guarantee product safety – challenges for N-of-1-treatments
Monday, February 24, 2025 at 12:30 pm US EST The traditional analytical life cycle for quality control of drugs fits not well with the...
385 views
Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
January 13, 2025 The presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children...
273 views
Personalized medicine for rare neurogenetic disorders: can we make it happen?
Agnies M van Eeghen , Hilgo Bruining , Nicole I Wolf , Arthur A Bergen , Riekelt H Houtkooper , Mieke M van Haelst , Clara D van...
64 views
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...
121 views
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