Researching the rare epilepsy CDKL5 – with Dr. Omar Mamad – FutureNeuro – RCSI – Ireland

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Ultragenyx update, May 2021

[vc_row][vc_column][vc_custom_heading text=”Ultragenyx update, May 2021″ use_theme_fonts=”yes”][/vc_column][/vc_row][vc_row][vc_column][vc_column_text]May 26, 2021

Dear Families Living with CDKL5 Deficiency Disorder,

While some of you may already know our company from previous presentations at scientific meetings, we are now pleased to provide your community with a more formal communication regarding Ultragenyx’s research on CDKL5 deficiency disorder (CDD).

Ultragenyx is currently in the early, pre-clinical stage of development for UX055, our investigational gene therapy program for CDD. Our investigational gene therapy is designed to deliver a fully functioning copy of the human CDKL5 gene to neurons. The goal is for those neurons to read the new gene and produce functional CDKL5 protein. We are using Adeno-Associated Vector Serotype 9 (AAV9) to transport the CDKL5 gene into neurons in the brain.

Click below to read the entire newsletter…..[/vc_column_text][ult_buttons btn_title=”Open Newsletter” btn_link=”|target:_blank|rel:nofollow” btn_bg_color=”#99003f” btn_bg_color_hover=”#981a4e” btn_title_color_hover=”#ffffff” icon_size=”32″ btn_icon_pos=”ubtn-sep-icon-at-left”][/vc_column][/vc_row]

CDKL5 International family conference

[vc_row][vc_column width=”1/2″][vc_single_image image=”799″ img_size=”large”][/vc_column][vc_column width=”1/2″][vc_custom_heading text=”CDKL5 Alliance International Virtual Family Conference” use_theme_fonts=”yes”][vc_column_text]The CDKL5 Alliance is pleased to present this year’s International Virtual Family Conference on Saturday, June 19th, 2021. The conference will be ‘a la carte’, meaning you will be able to visit the website on the 19th or afterwards and view pre-recorded presentations from the speakers outlined in the agenda below. You can watch any or all of the presentations in any order you like and will have the luxury of re-visiting the presentations in the future.

All presentations will be in English with subtitles available in Spanish, Italian, French, German, Russian and Japanese. You will have the opportunity to submit questions after watching these presentations on the 19th and answers will be posted within 2-3 weeks on the Alliance website.[/vc_column_text][/vc_column][/vc_row]