Share Price, page-2180

From the Phase 2 paediatric study of trofinetide - the RTT-DSC was one of the scales used. This same scale was not used in the Phase 3 study.


https://n.neurology.org/content/92/16/e1912


And then there’s NNZ-2591!

NNZ-2591 was tested in the ube3a knockout mouse model, which resembles features of Angelman syndrome in humans and includes motor deficits, learning problems and alterations in synaptic connectivity and plasticity. The study compared normal mice and "knockout mice" with a disrupted gene. In the knockout mice, treatment with NNZ-2591 for 6 weeks normalized the deficits in all the tests of anxiety, daily living, sociability, motor performance and cognition as well as eliminating seizures.

https://www.neurenpharma.com/irm/content/nnz-2591-in-angelman-syndrome.aspx?RID=481


NNZ-2591 was tested in the shank3 knockout mouse model of PMS, with treatment for 6 weeks. The study compared normal mice (“wild type” and mice with a disrupted shank3 gene (“knockout”. In the knockout mice, all behavioral deficits were restored to the wild type and treated knockout mice also showed a 83% reduction in susceptibility to seizures

https://www.neurenpharma.com/irm/content/nnz-2591-in-phelan-mcdermid-syndrome.aspx?RID=480