Research

All I Want for the Holidays Is Gene Therapy Research!

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CCDS Gene Therapy Research

Fundraiser Overview

This holiday season, help us bring Gene Therapy research to the creatine deficiency community! The 2019 ACD Holiday Heroes campaign aims to raise $50,000 to fund the first stages of CTD and GAMT deficiency gene therapy research.

We invite you to join us in our fundraising efforts as we strive to support research with the potential of improving the lives of CCDS patients. We’ve already raised $25,000 specifically earmarked for research thanks to generous donations and volunteer matches from friends, families, and employees from companies like Microsoft and Google.

To help guide our efforts in supporting gene therapy,  the ACD has formed a CCDS Gene Therapy Consortium, an international collaboration between CCDS researchers, gene therapy experts, and stakeholders interested in developing gene therapy for creatine deficiencies. The mission of the consortium is to explore the feasibility of gene therapy for Creatine Transporter Deficiency, as well as GAMT Deficiency, and to promote the development of gene therapy programs in multiple laboratories with the end goal of sharing data and reaching clinical trials as quickly as possible. 

Current CCDS Gene Therapy Consortium members include:

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Get Involved and Show Your Support!

  • Donate to Support CCDS Gene Therapy Research: Get started by clicking here.
  • Create a Facebook Fundraiser Supporting the ACD: Create a Facebook Fundraiser and share with your friends and family why supporting CCDS Gene Therapy Research is important to you! Creating a fundraiser is easy, get started by clicking here.
  • Order CCDS Pins + Educational Postcard: Give the gift of CCDS awareness this holiday season and order CCDS Pins and Postcards. These “donation on your behalf” gifts are great for teachers, neighbors, family, doctors, and more! Click here to get started.