Clinical Trial Readiness Educational Series
We believe that helping educate our families about drug discovery and clinical trials is essential to having an informed community that can consent to participate in studies. In September of this year, we will launch an educational series to engage the SCN8A families in important discussions surrounding drug discovery and clinical trials. Check out the sessions schedule below. Videos of the series sessions will be posted below as they become available.
Part 1—Humanizing Language in Pediatric Healthcare
The Awesomeness Form with Rachel Callander.
To kick off our five-part SCN8A Clinical Trial Readiness Series we have writer, advocate, photographer, and mother Rachel Callander leading a workshop with us focused on how we talk about our children. In this first event, we will look at Rachel’s Awesomeness Form, which is a take on a developmental milestones form, and talk about how to fill one out for each of our children, to celebrate their uniqueness, their talents, their abilities. So that as we look with hope toward new therapies in our future, we also take time to pause and remember the beauty at the center of that hope.
To kick off our five-part SCN8A Clinical Trial Readiness Series we have writer, advocate, photographer, and mother Rachel Callander leading a workshop with us focused on how we talk about our children. In this first event, we will look at Rachel’s Awesomeness Form, which is a take on a developmental milestones form, and talk about how to fill one out for each of our children, to celebrate their uniqueness, their talents, their abilities. So that as we look with hope toward new therapies in our future, we also take time to pause and remember the beauty at the center of that hope.
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Part 2—A Conversation About Patient Data
Gabi Belfort, MD PhD, Praxis Precision Medicines, John Schreiber, MD, Children's National, Vanessa Vogel-Farley, DUP15Q Alliance and RareX, and Hillary Savoie, PhD, Founder/Director The Cute Syndrome Foundation
A conversation with clinical, industry, and advocacy leaders about the value, uses, and ethics of patient data collection.
A conversation with clinical, industry, and advocacy leaders about the value, uses, and ethics of patient data collection.
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Part 3—Clinical Trials 101
Carolyn McMicken, PhD, Neurocrine Biosciences, John Millichap, MD, Lurie Children's, Luke Rosen, Ovid Therapeutics, and Hillary Savoie, PhD, Founder/Director The Cute Syndrome Foundation
Understanding clinical trial design, inclusion/exclusion criteria, outcome measures, and timelines with the help of clinicians, advocates, and industry.
Understanding clinical trial design, inclusion/exclusion criteria, outcome measures, and timelines with the help of clinicians, advocates, and industry.
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Part 4—Setbacks and Failures in Drug Development
Bernard Ravina, MD, Praxis Precision Medicines, Gail Farfel, PhD, Zogenix, JP Johnson, PhD, Xenon Pharmaceuticals, and Hillary Savoie, PhD, Founder/Director The Cute Syndrome Foundation
A conversation with industry about the challenges of drug development and how they plan for them.
A conversation with industry about the challenges of drug development and how they plan for them.
Click below to view the slides from this presentation.
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