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 and for recruitment to clinical trials - paper (2.91 MB)