Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

NTSAD Webinars

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

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?

Glossary of Terms here.

Wednesday, August 7, 2019

IntraBio and IB1001 Clinical Trials 


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.

pdf Acetyl-DL-leucine in Niemann-Pick (Type C) - paper (587 KB)

pdf Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C  - paper (2.91 MB)

Watch the webinar here


Is there a particular topic you'd like to see discussed in a webinar format? Feel free to send an email to NTSAD This email address is being protected from spambots. You need JavaScript enabled to view it..

Upcoming Webinars

Wednesday, April 1, 2020
2:00 pm (Eastern)
11:00 am (Pacific)
Taking Care of You: Self-Care in Challenging Times


Are you searching for manageable ways to take some time to care for yourself especially during moments of stress and uncertainty?

Please join National Niemann-Pick Disease Foundation (NNPDF) and NTSAD as we jointly present the upcoming forum Taking Care of You: Self-Care in Challenging Times led by Shoshanna Hecht, LCSW, on Wednesday, April 1, 2020 at 2:00 pm EST/11:00 am PST via Zoom Webinar.

This community forum will include practical tips on manageable, every day self-care, a guided meditation, and time for Q&A.

Shoshanna Hecht is an Executive and Personal Coach based in New York City, with previous experience of over 10 years as a clinical therapist on the west coast. Shoshanna also leads guided meditations to boost confidence while reducing stress, self-doubt, and overwhelm. They can be found both on her website and on the Meditation Studio App named one of Apple’s 10 Best Apps of the Year.