Day 1 - Part 1
Welcome and Opening (8:18 - 18:20) | Glenn and Cara O’Neill [Co-Founders and President and Chief Science Officer, respectively, Cure Sanfilippo Foundation | Parents of Child with Sanfilippo Type A]
“Research Happening Now” Session (Part 1) Targeting Heparan Sulfate (19:55 - 1:26:50) | Luigi Pavone [Professor Biochemistry, Molecular Medicine, and Medical Biotechnology at University of Naples Federico II]; Karl Johe [Chief Executive Officer of ReMotorTherapeutics]; Diego Medina [Cell Biology and Disease Mechanisms at Telethon Institute of Genetics and Medicine (TIGEM)]; and Antonella Capuozzo and Giancarlo Parenti [Telethon Institute of Genetics and Medicine (TIGEM)] joined for Q&A
Dopamine, Autism, and Applied Behavior Analysis (ABA) in Sanfilippo [1:26:51 - 2:16:06) | Elvria DeLeonibus [Group Leader, Neurogenetics, at Telethon Institute of Genetics and Medicine (TIGEM)]; and Suzanne Moore [Clinical Director at Bardin Behavioral Health]
Connecting Life & Science: Sanfilippo Type C (2:16:10 - 3:06:28) | Christi Ormeroid, Cybill Aros-Pearson, and Marisa DiChiacchio [parents of children with Sanfilippo Type C]; Lauren Dengle [Assistant Professor, Pediatrics and Neurology & Neurotherapeutics at UT Southwestern]; and Rafael Badell-Graum [Post Doctoral Fellow, BioMedical Research Facility, at University of California, San Diego]
Lunch & Learn: Advancing Newborn Screening (3:06:31 - 3:32:06) | Matthew Ellinwood [Chief Scientific Officer for National MPS Society]; and Melissa Wasserstein [Professor of Pediatrics and Genetic Medicine at Albert Einstein School of Medicine]
24 окт 2022