Ten years ago, neither Allyson Berent, Terry Pirovolakis nor Michelle Werner expected to be founders of biotech companies. But they’ve all done so with the goal of helping to treat their children’s rare diseases.
The three parents took the stage at the 2024 STAT Summit to talk about the challenges of trying to find treatments for rare diseases — from securing funding to translating research in animals to humans to commercial barriers.
“What we realized was that if we didn’t continue to do this ourselves, nobody was going to do it, because the priorities of parents and patients will never change. We have to wake up every morning and go to bed every night and promise that little girl that I’m going to make her life better,” said Allyson Berent, chief scientific officer at the Foundation for Angelman Syndrome Therapeutics. Angelman syndrome is a neurogenetic disorder that occurs in 1 in 15,000 births.
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