Clinical Trial Readiness
Fall 2022

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Part 1–From Data to Development: A Deeper Look at PRAX-562 and Your Role in its Progress

Kalley Dalby, Raji Mahalingam, Christian Rubio, Karen Utley, Praxis, and Kacie Craig, The Cute Syndrome Foundation
We kicked off The Cute Syndrome Foundation’s 3rd Annual Clinical Trial Readiness Series with a live community conversation with Praxis Precision Medicines to discuss the molecule PRAX-562 and its potential for SCN8A.

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Part 2–Research Updates and Moving Beyond Clinical Trials 101

Heather Bentley, Carolyn McMicken, Tyler Reagan, Svetlana Shore, Neurocrine, and Kacie Craig, The Cute Syndrome Foundation
For week 2, we welcomed back Neurocrine to discuss NBI-921352 and Kayak Study updates, with a look back at the last 6 months and whats to come in the next 6 months of trial. We also moved beyond Clinical Trials 101 to learn more about select clinical trial roles and responsibilities and the importance of treatment blinding and placebo effect.

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Part 3–The Cute Syndrome Foundation Updates and Drivers

Kacie Craig, Shelley Frappier, and Karen Varner, The Cute Syndrome Foundation
The final part of the 2022 Clinical Trial Readiness series was a casual conversation with members of TCSF's executive committee to discuss updates on the Neurocrine Biosciences lab tour and the Global Genes Summit conference, as well as provide an overview of the 8th Annual SCN8A Clinician, Researcher, and Family Gathering! The discussion also included foundation drivers and aspirations for TCSF’s future.

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