The kiddo in the above photo is named Paxton. He desperately needs your help!
I learned from his dad that Paxton has a very rare genetic mutation. He has a devastating neurodegenerative mutation on the CLCN6 gene. Paxton will be part of a clinical trial for a promising new lifesaving treatment, but funds need to be raised.
Scientists at UMass Worcester, Chan School of Medicine, Horae Gene Therapy Center, are developing a first of its kind gene therapy for CLCN6 mutations. Paxton will be the patient in the clinical trial.…