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

The 4th Annual Gene Therapy for Rare Disorders Europe is dedicated to overcoming manufacturing, clinical, and commercialization challenges drug developers face when delivering gene therapies to market. Considerations for Selection of NovelEndpoints and Appropriate Controls • Discussing some of the creative manners gene therapy developers have used to optimise clinical trial design • Collecting the […]