Email
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Hope for Hasti
Virtual Scientific Conference - Agenda
*All times are in Eastern Time (ET)
9:00 am - Conference starts - introduction to the day
9:15 am - Dr Angelo Selicorni - MRI Findings in CdLS patients
9:40 am - Dr Matthew Deardorff, MD
Director of Translational Genomics, Director of Personalized Care, Children's Hospital of Los Angeles
Genetics of the CdLS HDAC8 mutation
10:05 am - Dr Valentina Massa
Associate Professor in Applied Biology, University of Milan
Lithium's beneficial effects in CdLS experimental models
10:30 am - Dr Cat Lutz, Ph.D, MBA
Director of the Mouse Repository and the Rare and Orphan Disease Centre at The Jackson Laboratory
JAX Rare and Orphan Disease Centre
10:55 am - Dr Adriano Flora Ph.D
Senior Manager, Business Development at The Jackson Laboratory
Preclinical Program for Hope for Hasti
11:15 - 11:25 - Break
11:25 am - Aamir Zubeiri Ph.D
Technology and Resource Development Scientist, Rare and Orphan Disease Program, The Jackson Laboratory
Basic strategies for developing mouse models and preclinical drug testing for rare diseases
11:50 am - Dr Dolan Sondhi Ph.D
Professor of Research, Genetic Medicine, Associate Director Gene Therapy Core Facility, Weill Cornell
New drug clinical trial for neurological diseases in children
12:15 pm - Dr Stuart Cobb
Head of translational research laboratory, Simons Fellow and Reader in Neuroscience at the University of Edinburgh.
AAV9 as a platform type of approach toward a treatment
12:40 pm - Dr Neil Hackett Ph.D
Transition from mouse to clinical trial
1:00 pm - 1:45 pm - Break for lunch
1:45 pm - N of 1 clinical trial panel session
Rafael Scandon, Andrew McFadyen and Lisa Kearns from the Paediatric Gene Therapy Medical Ethics (PGTME) Working Group
2:10 pm - Chris Brannigan
Founder, CdLS Hope for Hasti
CdLS, Our family's story
2:35 pm - Gail Rich
Founder, The Nicole & Jessica Rich Foundation
The difference a treatment makes
3:00 pm - Q&A session for families
3:30 pm - Conference ends
Download a pdf copy of the agenda
