ELJ-101: Your Guide to the Gene Therapy Webinar Highlights!
On Rare Disease Day, we gathered as a global CDKL5 community to watch the ELJ‑101 gene therapy webinar, presented by Russ Addis, PhD, Chief of Pipeline Strategy and Head of Genetic Medicine at the LouLou Foundation.
The webinar marked an important moment. It offered a clear update on where the ELJ‑101 gene therapy program stands, what researchers are learning, and what the path forward may look like. Gene therapy is complex, and this kind of information can be difficult to absorb — especially when it directly relates to our children and loved ones. Russ did an excellent job explaining the science thoughtfully and transparently, but we also recognize that this material can still feel overwhelming, emotional, or hard to fully process in one sitting.
That’s why, following the webinar, we created additional resources to help families better understand the information at their own pace, in formats that meet different needs.
Watch the ELJ‑101 Webinar
If you haven’t watched the webinar yet — or would like to revisit it — you can view the full recording here! The webinar covers:
- The scientific rationale behind ELJ‑101
- What preclinical studies have shown so far
- Why early trials focus on safety
👉 WATCH THE ELJ‑101 WEBINAR VIDEO – HERE
Hope on the Horizon: A Simple Guide to the ELJ‑101 Gene Therapy Webinar
We created a warm, heart led resource for families who want a simpler entry point into the information. This guide is designed for parents, caregivers, and adult family members who want:
- An accessible explanation of gene therapy
- A high‑level recap of the webinar
- Clear language without unnecessary technical detail
- A resource you can return to when you’re ready
👉 Family Friendly Easy Read- Link Here
The Super‑Science Helper: A Guide to ELJ‑101 for Siblings & Kids
Many families also asked how to talk with siblings and children about what they heard, felt, or sensed after the webinar. To support those conversations, we created a resource just for them. This child‑friendly guide:
- Explains CDKL5 and gene therapy using familiar ideas
- Helps kids understand why science takes time
- Reassures siblings that they are an important part of their family’s journey
A Detailed Plain‑English Summary of the Webinar
For families who want a more detailed summary we are sharing a comprehensive plain‑English summary.
👉Traditional Plain English Summary
Walking This Path Together
Gene therapy research moves forward carefully, step by step. Families live with CDKL5 every day. Our goal with this webinar — and with these additional resources — is to help bridge that gap with clarity, honesty, and care.
All videos in the From Science to Hope: Walking the Path toward CDKL5 Gene Therapy series will continue to be shared, along with new resources as information becomes available.
Thank you for taking the time to learn, reflect, and walk this path with us — one thoughtful step at a time.