Tuesday, March 31, 2020

COVID-19 and its impact on
the rare disease community

Panelists:

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?

Presenter:

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

Presenter:

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?

Glossary of Terms here.

Wednesday, August 7, 2019

IntraBio and IB1001 Clinical Trials 

Panelists:

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.