2024 GNAO1 Conference | The Bow Foundation

2024 Conference Photos

AAV9 gene therapy for GNAO1 deficiency
Miguel Sena Esteves, PhD

Automated home based assessment of dystonia in children with GNAO1
Larissa Heideman, MD

Broadening of phenotypic spectrum and new treatment options in GNAO1
Moritz Thiel, MD

Developing a CRISPR-mediated gene therapy for GNAO1 Encephalopathy
Pardis Kazemian, PhD Candidate

Epilepsy in GNAO1
Erika Takle Axeen, MD

European Natural History Study and search for novel biomarkers in GNAO1
Giovanna Zorzi, MD

GNAO1 mutations: molecular insights, disease modelling, and therapeutic developments
Vladimir L. Katanaev, PhD

GNAO1 Natural History Study Update
Amy Viehoever, MD, PhD

Inclusive Education Panel: Voices of Experience

Learning the Dance of our Child
Penny Graham, B.S.W, GNAO1 mother, advocate

Lipid nanoparticle mediated delivery of CRISPR gene therapy for neurologic disease
Blair R. Leavitt, MDCM

Molecular annotation of G protein variants in GNAO1 disorder
Lizzy Obarow, PhD Candidate

Mouse model of dominant negative GNAO1 disorder
Gloria Brunori, PhD

Pathogenetic mechanisms and potential therapies: Insights from Italian studies on animal models and stem cells
Simone Martinelli, PhD

Rare Disease Treatment One Patient at a Time: Anti-Sense Oligonucleotide Therapy for GNAO1-Associated Disorder
Jennifer Friedman, MD

Supporting family wellbeing
Meri Viano, PPAL Associate Director

The alpha and beta gamma of GNAO1 signaling
Terry Hébert Professor

The effect of GNAO1-associated disorder variants on iPSC-derived neural cells
Harald Mikkers, PhD

The severity puzzle: unlocking insights into GNAO1-related disorders
Juan Darío Ortigoza-Escobar, MD, PhD

Tips and Tricks for Leveraging Technology for Communication and Connections with our Complex Communicators
Hillary Jellison, M.S. CCC-SLP, ATP