Ultragenyx’s research on CDKL5 deficiency disorder – May 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,…

Summary of CDKL5 research from 2020.

We would like to share with you an excellent review of the research in CDKL5 of the last 12 months written by our Trustee & Dad of Ellie Martyn Newey – www.supporting-cdkl5.co.uk. You are able to find the summary of the research in 2020 of CDKL5 Martyn Newey by using the following link  

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…

Rett Disorders Alliance

Some good news to share which we will hope will change the face of Rett and Rett-like disorders within the health and social care sectors in the UK. A collective voice creates stronger communities ‪#‎togetherwearestronger ‬‪#‎curecdkl5‬ Therefore we are delighted to announce the formation of the Rett Disorders Alliance of the UK. The Rett Disorders Alliance…