International Virtual Family Conference

This year the CDKL5 Alliance is pleased to present the International Virtual Family Conference on Saturday, which regretfully we are not able to host the event in person.

Therefore we have chosen to make the conference will be ‘a la carte’ in the sense that you will be able to visit the cdkl5alliance.org website at any time to watch the pre-recorded presentations from the speakers outlined in the agenda. You can watch any or all of the presentations and will have the luxury of re-visiting these presentations in the future.

Most presentations will be in English with subtitles available in Spanish, Italian, French, German, Russian, Japanese and many more languages by using the YouTube translation option if available. How to do this please find more information by clicking here.

You will have the opportunity to ask questions to the speakers by using our online question form after watching these presentations on the 19th or later date. We will aim to have the answers or most of them posted on our website within 2-3 weeks or where needed answered by mail to the e-mail you provided.

*This page will be available for 2 to 3 months, after this it will close – however, all video’s will still be available on our youtube channel.

Patric Benz

Welcome & Introduction

Prof. John Rouse

Investigating CDKL5 function and the rebalancing of CDKL5 deficiency disorders

Prof. Elisabetta Ciani

Innovative Strategy to enhance the efficiency and safety of gene therapy for CDD

Prof. Ralf Schmid

Recent research advances in developing a CDKL5 gene therapy

Prof. D. Battaglia

Epilepsy management in CDD

Prof. Tim Benke

Symptom management in CDD: A to Z

Dr. Xavier Liogier d’Arduy

How to optimize drug development for CDD?

Dr. Helen Leonard

“The International CDKL5 Disorder Database (ICDD): stepping from the past into the future”

Dr. Femida Gwadry-Sridhar

What is an International Registry and why is it important?

Robyn Allen

Hippotherapy: an effective therapeutic tool for children with CDKL5

Carol-Anne Partridge

It doesn’t just affect me - understanding impact of trauma, a family perspective

Reini Linder

Skiing experience for CDKL5 children with the “Dualski”

Zogenix, Torbjorn Waerner

Zogenix: our commitment to developing new treatments for Patients living with rare diseases.

Ultragenyx, Sharyl Fyffe-Maricich

Preclinical Evaluation of AAV9-CDKL5 Gene Therapy in Models of CDKL5 Deficiency Disorder

Marinus, Alex Aimetti

Investigational Drug Ganaxolone for CDKL5 Deficiency Disorder – Advancing the Treatment Paradigm

Amicus, Brian Ranes

Preclinical drug development: an overview

Natalie Ladly

General remarks CDKL5 Alliance

Antonino Caridi

Organization Running

Carol-Anne Partridge

CDKL5 Alliance Media

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