Cure CLCN6

Join us on our rare disease journey, as we blaze a trail to the new frontier of medicine: Gene Therapy.

Our son Paxton was diagnosed with an ultra rare genetic mutation on his CLCN6 gene. The odds of having a mutation on this gene are about 1 in a 1,000,000 (one in a million). His mutation is known to be neurodegenerative in nature and affects every aspect of his life. He has severe epilepsy, autism spectrum disorder, hypotonia, severe developmental delay, poor motor coordination and apraxia of speech. 

This channel will follow the daily life of Paxton and our family, the challenges he faces and the progress he makes, as we work with University of Massachusetts Worcester, Chan School of Medicine, Horae Gene Therapy Center, to develop a gene therapy and clinical trial for him.

We would love your support as we venture into uncharted territory and hold out hope for a better future for Paxton than we ever imagined.