Leading the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases
NTSAD officially has launched a webinar series to bring the experts to the community to discuss topics ranging from research advances to issues that impact the family.
Tuesday, March 31, 2020
COVID-19 and its impact on the rare disease community
Staci Kallish, DO Cynthia Tifft, MD, PhD Camilo Toro, MD
Dr. Kallish, Dr. Tifft and Dr. Toro will discuss and answer questions about how COVID-19 impacts the lives of children and adults affected by rare diseases.
Thursday, January 23, 2020
Who is the FDA and what do they do?
Curtis Scribner, MD Independent Biotech Pharma Regulatory and Clinical Consultant
The goal of this webinar is to inform the NTSAD community about the regulatory side of clinical trials. We are grateful to Dr. Curt Scribner for lending his time and sharing his expertise with the NTSAD community to shed light on the what, how and why behind the process of a clinical trial's approval.
Friday, December 6, 2019
What is a Clinical Trial: Demystifying the Process
Gerald Cox, MD, PhD NTSAD Board Member Boston Children's Hospital
Outline: What is a clinical trial and what do they involve? What are the different types of clinical trials? Who oversees clinical trials? FDA oversees each step of the drug development How do I know if I or my child is eligible for a clinical trial? How much does it cost to participate in a clinical trial?
Taylor Fields, Senior VP, IntraBio Tatiana Bremova, MD, PhD, University Hospital Bern, Switzerland
A presentation about IntraBio's compound, IB1001, the clinical trials in the US and Europe, and the hope that it has a positive impact on the GM2 Gangliosidoses (Tay-Sachs and Sandhoff). Facilitated by Staci Kallish, DO, NTSAD Board president.