Help Save Paxton's Life: A Rare Genetic Battle

Paxton's Story: A Rare Genetic Battle

Our son Paxton, recently diagnosed with an ultra-rare genetic mutation on the CLCN6 gene, faces immense challenges including Autism Spectrum Disorder, severe epilepsy, hypotonia, and poor motor coordination. This mutation is neurodegenerative, meaning time is critical.

The Urgency of Paxton's Condition

Paxton is one of only 10 known cases worldwide, and currently, no research exists for a gene therapy due to the small patient population. However, we have a unique opportunity to develop a life-saving treatment for Paxton at the University of Massachusetts' Horae Gene Therapy Center.

Why We Need Your Help

The scientists will create a custom gene therapy using specialized viruses called Adeno-Associated Viruses (AAV). Each AAV costs 1.5 million to produce. Due to Paxton's specific needs, two AAV's are required, totaling $3 million. The entire process, including studying the mutation in laboratory models, developing and testing the therapy, and administering it to Paxton, will take approximately three years.

The Science Behind the Therapy

The University of Massachusetts' Horae Gene Therapy Center will develop, test and deliver a gene therapy for Paxton through a clinical trial. This therapy involves using specially designed viruses to replace the faulty gene with a functional one.

Funding Breakdown and Timeline

We need to raise these funds urgently to give Paxton the chance he deserves. Your support will directly fund the development of these crucial AAV's. Time is against us, but with your help, we can make this happen.

How You Can Make a Difference

From the depths of our hearts, thank you to everyone who reads Paxton's story and donates. Please share our campaign to reach as many people as possible. Every dollar counts towards saving our son's life.

Follow Paxton's Journey

Follow our journey and get updates through our website and YouTube channel. More social media links will be available soon.

Website: https://cureclcn6.godaddysites.com/

YouTube Channel: http://www.youtube.com/@CureCLCN6

On Facebook, Instagram and TikTok follow Paxton's Pioneers

Heartfelt Thanks and How to Share

From the bottom of our hearts, thank you to everyone who has read this story and has donated to help our son. Please share our story with others to help us reach a wider audience and to reach our goal. 100% of all donations will go towards developing the AAV's necessary for Paxton's gene therapy.