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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 wrote.
"He'll develop movement disorders and seizures, suffer severe dementia, endure a lot of pain and suffering, and then he'll die. All in the coming years."
Over $896,000 in funds have been provided to the GoFundMe that would kickstart the first-ever gene therapy clinical trial for Connor's type of Sanfilippo syndrome.
Their goal is to raise $3 million to fund the trial, which in pre-clinical models shows that it can stop the disease.
There is no guarantee that Connor will be among the children accepted into the trial, but his parents believe this is their only hope.
"We can't just sit back and accept that he's going to fade away and die, not when there is something that can save him," DiChiacchio wrote.
Click here to donate to "Save Connor."
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