RAREsummit23 poster gallery
Meet the RAREsummit23 poster exhibitors who’ll be showcasing research and patient journey posters at the event.
Patient Registries at the JWMDRC: where we are now and where we might go next
Researcher-Caregiver Engagement in Lowe Syndrome and Dent Disease
Drug repurposing to treat the rare genetic disease alkaptonuria (AKU) – The AKU Society
Living with Noonan Syndrome – Loughborough University & The Noonan Syndrom Association
CACNA1C-related disorders (CRD) & Timothy Syndrome Alliance (TSA)
Galactosialidosis disorder and path to gene therapy/cure
Developing a Decision-Making Framework for Expanded Access to Gene Therapy in Rare Neuromuscular Diseases
