Roche Pharmaceuticals is exploring an investigational drug called Alogabat to address the deficiency of GABA expression (GABAA α5 receptors) in deletion positive individuals living with Angelman syndrome.
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A small molecule that is specific to individuals living with deletion+ AS aimed to improve the function of the GABAA alpha5 receptors, which are made by another gene missing in individuals with deletions. This protein is important brain function, a GABA neurotransmitter, and the reduced amount in those living with deletions may exacerbate their challenges.
Alogabat
Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. (Aldebaran)
Deletion
ALDEBARAN
