Boston Family & Researcher Gathering - August 2026

Family Meetings & Events
Written By Teri De Clercq

In August 2026, families, researchers, and clinicians came together in Boston for our latest Family & Researcher Gathering on NARS1 Disorder. Alongside time to connect as a community, the weekend brought research updates spanning drug repurposing, pre-clinical models, biomarkers, and ASO/gene therapy, as well as practical sessions on registries, caregiver resources, and fundraising. Watch the sessions below.

Day 1

Welcome & Family Introductions

 

Conceptos básicos de NARS1 con Ingrid

 

Introduction to pre-clinical models with Ingrid

 

Reutilización de drogas con Rachel

 

Therapeutic Rescue of NARS1 Variants with Jenica Kakadia, Western University, Canada

 

Biomarkers with Luc Legon, Amsterdam, UMC

 

Mouse Models with Rob Burgess from Jackson Laboratory, USA

 

ASO/Gene Therapy - Tools for Clinical Trial Readiness with Matthew Fuller, Ultragenyx

 

Fireside Chat: Measurement Science with Rachel and Natasha Ludwig, Kennedy Krieger Institute

 

Day 2

NARS1 Disorder - What we know from Rare-X data with Anastasia Yankovskiy, Children Hospital of Philadelphia

 

How to use Rare-X with Teri

 

Caregiver Resources with Cat

 

Fundraising & Conference Close with Teri

Teri De Clercq
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Drug Repurposing, Explained