STAT

Scientists see hope in a rare disease drug Wall Street has all but written off

At the nation’s biggest neurology conference, scientists endorsed Ovid’s data on an experimental drug to treat Angelman syndome.

Amanda Moore can still smell the room.

It was Jan. 26, 2016, and she was standing in a doctor’s office, listening to an explanation of why Jackson, her smiley toddler, might never walk, talk, or sleep through the night. He had Angelman syndrome, a rare genetic disorder that causes seizures and severe cognitive impairment. There is no treatment.

“When you’re first diagnosed, the dream you had for your child kind of dies,” Moore said. “But you have to pick yourself back up and start living out the new dream for your child.”

That dream compared to those on placebo. It was a small trial, enrolling 88 patients, but it hinted at an unprecedented therapy that could change Jackson’s life.

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