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Gene Therapy

Community Rallies For Philly Area Boy With Childhood Alzheimer's Community Rallies For Philly Area Boy With Childhood Alzheimer's
Community Rallies For Philly Area Boy With Childhood Alzheimer's Support is surging for a 13-year-old Chester County boy who is fighting a rare, degenerative syndrome that has been compared to Alzheimer’s. Connor Dobbyn was recently diagnosed with Sanfilippo syndrome Type C, a fatal brain disorder caused by a single missing enzyme. "Because Connor lacks this single enzyme, toxic storage material is building up in his brain every second and damaging it," Marisa DiChiacchio, his mom, wrote on a GoFundMe. There is currently no FDA-approved treatment or cure. Connor will eventually lose the ability to walk, talk, and feed himself, DiChiacchio wro…