Community Experts Discuss a Range of Topics – From Research Advances to Family Issues

Recognizing Seizures

Dr. Jeffrey Buchhalter, a neurologist NTSAD connected to through the Child Neurology Foundation, met with a group of families to discuss seizures, what they are, how they can present, and the issues related to them.

Moderated by Becky Benson, NTSAD Family Services Manager

Note: Families in this video gave permission to be recorded and have their questions shared publicly.

Choosing Resiliency in the Face of Adversity

Sheryl Sandberg, Facebook COO, founder of Lean In, and co-author of Option B: Facing Adversity, Building Resilience, and Finding Joy, joined Blyth Lord, Founder of Courageous Parents Network, in a conversation about the challenges they faced and what they learned following unexpected, life-altering situations. Sheryl and Blyth along with Becky Benson, NTSAD’s Family Services Manager and mom to Miss Elliott, discussed anticipatory grief, the importance of being mindful of things that bring you joy even during hard times, and leveraging post-traumatic growth.

More than 200 people participated in this poignant and inspiring conversation filled with personal stories and examples of how to build resiliency and support others through difficult experiences.

COVID-19 and Its Impact on the Rare Disease Community

A panel of rare disease experts discuss and answer questions about how COVID-19 impacts the lives of children and adults affected by rare diseases.


  • Staci Kallish, DO
  • Cynthia Tifft, MD, PhD
  • Camilo Toro, MD

What is the FDA, and What Is Its Role in the Clinical Trial Process?

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.


Curtis Scribner, MD
Independent Biotech Pharma Regulatory and Clinical Consultant


Susan Kahn, former NTSAD Executive Director

Defining a Clinical Trial


  • What are clinical trials and what do they involve?
  • What are the different types of clinical trials?
  • Who oversees clinical trials?
  • How does the FDA oversee 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?


Gerald Cox, MD, PhD
NTSAD Board Member, Boston Children’s Hospital

IntraBio and IB1001 Clinical Trials

Here is 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 diseases).

Facilitated by Staci Kallish, DO, former NTSAD Board President


  • Taylor Fields, Senior VP, IntraBio
  • Tatiana Bremova, MD, PhD, University Hospital Bern, Switzerland

Is there a particular topic you’d like to see discussed in a webinar format? Feel free to send an email to NTSAD here.