Ultragenyx update, May 2021

Ultragenyx update, May 2021 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…

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CDKL5 International family conference

CDKL5 Alliance International Virtual Family Conference 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 cdkl5alliance.org website on the 19th or afterwards and view pre-recorded presentations from the speakers outlined in the agenda below.…

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Twenty “must-read” publications from 2020

#1 to #3:  Gene therapy and X reactivation: Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder. Gao Y et al., Brain Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder. Benke TA and Kind PC, Brain First published proof of concept for AAV-mediated gene therapy for the treatment…

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Marinus Launches Expanded Access Program for Ganaxolone Treatment in CDKL5 Deficiency Disorder ​

Marinus Launches Expanded Access Program for Ganaxolone Treatment in CDKL5 Deficiency Disorder RADNOR, Pa.–(BUSINESS WIRE)– Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the launch of an expanded access program (EAP) that will allow the company to offer ganaxolone to patients…

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