Presentation of Samantha Parker, Chief Patient Acces Officer. https://www.youtube.com/watch?v=nuIoI8cqNfc

This symposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. Michaël Hocquemiller, Head of Non-Clinical Development, will present biomarker data from the ongoing AAVance clinical trial with […]

World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo Syndrome, a disease few have heard of. This special day of Awareness is in honor of the children around the world living with Sanfilippo Syndrome today, and those who have passed away. It also honors the families of these precious children. […]