Hope for PDCD Through Gene Therapy

April 20, 2023

We have an angel on earth, a little girl named Aela, who is battling PDCD, a rare and ultimately fatal metabolic genetic disease. Aela is six years old. She loves music, her favorite bongos and being outside. She has had some bumps in our journey to get to where we are today though. Aela’s first two years of life were very difficult. From having hundreds of seizures a day from infantile spasms, to searching for a diagnosis, all while having her dad deployed to Iraq…it was a very heavy time.

Fast forward to today. Finding her PDCD diagnosis through WGS and a plan of care has made a world of difference for Aela. She has eyes that look into your soul and a smile that lights up the world. While we are grateful for the ketogenic diet to slow the progression of PDCD, Aela, and all the children battling PDCD deserve the best science has to offer. In partnership with the Hope for PDCD Foundation, we are currently in phase 1 of gene therapy research and are hopeful for the future of children battling this awful disease. It will be a long and expensive journey, but one we must follow.

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