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. […]

Strategy, advocacy and partnering for the orphan drug industry World Orphan Drug Congress 2020 Panel: Optimising clinical development strategies for gene therapy. Full panel to be confirmed. Samantha Parker, Senior Vice President & Chief Patient Access Officer, Lysogene Mathew Pletcher, Head of Rare Diseases, Roche Christeen Moburg, Senior Director, Patient Advocacy, Sangamo Therapeutics