Current assessment tools are just not sufficiently sensitive or objective. You don't need to be an MD or a PhD to see that the data Emerald collected directly from an individual provides richer, more quantitative data than a questionnaire that asks parents or clinicians to interpret how their child or patient is doing.
Clinical trials would significantly benefit from assessment tools that are sensitive, accurate, and objective, and that focus on symptoms that are critical to our children’s well-being. Having tools like Emerald will help ensure that medicines being tested and approved for Rett provide meaningful improvements.
Of course, we hope that the upcoming gene replacement trials and all future genetic medicine trials will result in dramatic improvements that will be obvious to all with a simple before and after video. However, regardless of how dramatic the results are, they will take time to manifest.
Here are several reasons why having an objective assessment tool is important:
While biopharma companies see incredible value in having reliable assessment tools, they don’t have the time, resources, or bandwidth to develop them. It’s therefore crucial that RSRT addresses this gap to support Rett development programs.
The validation study will be recruiting patients from the Rett clinics at Boston Children’s Hospital and Rush University in Chicago. We will share more information as the study prepares to recruit.
I thank all of our families and supporters who make this study possible and join us in our mission to cure Rett.
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