THIS IS IN DRAFT
Webinar Outline
Introduction (5 minutes):
To be presented by the AGSAA
Welcome and introductions
Importance of clinical trials for the development of new medicines
Brief introduction and handoff for Andy and Transposon
Transposon overview (10 minutes):
To be presented by Transponson’s Medical Director
Overview of Transposon's history and mission
Explanation of their new medicine and how it works
Brief summary of the preclinical research that led to the clinical trial
Explanation of available safety data, including any preclinical safety studies and early-phase clinical trials that have been conducted to assess the safety and tolerability of the medicine
Highlight any compensation or incentives that will be provided
Share Transposon’s plan to keep families informed of trial developments
Trial procedures (15 minutes):
To be presented by Site Investigators.
Introduction of the site investigators (AGS doctors) who will explain the trial procedures
Overview of the inclusion and exclusion criteria for the trial
Discussion of the trial design, including the number of participants and the length of the trial
Detailed explanation of the trial procedures by the site investigators, including the medication dosage and frequency, the study visits, and any tests or assessments that will be performed
Discussion of the potential risks and benefits of participating in the trial from the clinician’s perspective
Conclusion (5 minutes):
To be presented by the AGSAA
Recap of key points
Q&A session to address any remaining questions or concerns
Information about how to contact Transposon or the site investigators for further information or to participate in the trial
Highlight AGS patient registry and AGSAA contact list as ways to stay up to date