Amicus Therapeutics (NASDAQ:FOLD) perks up 2% premarket on light volume in response to encouraging preliminary data from a Phase 1/2 clinical trial evaluating its AAV-CLN6 gene therapy in patients with a rare inherited nervous system disorder called CLN6 Batten disease.
Interim data from the first eight children receiving a one-time intrathecal (into the spinal canal) administration of AAV-CLN6 showed a disease stabilization rate of 88% (n=7/8) up to two years after treatment.
The safety profile was favorable with most adverse events being mild.
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