The Inborn Errors of Metabolism Drug Development Summit 2020 is a conference focused on key patient access, regulatory, clinical and commercial aspects of gene therapy.
Topics
- A New Era in Cellular Medicine for Phenylketonuria
- Development of an Oral, Non-Systemically Acting Medicinal Product for the Treatment of Phenylketonuria (Pku) to Be Used as a Mono-Therapy
- Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases
- Treating PKU by Lentiviral Vector Gene Delivery to Liver
- Delivering on Hope for Patients with Serious Chronic Diseases
- Development of Leriglitazone in Rare Neurodegenerative Diseases
- Early Commercial Strategy for Rare Indications
- Patient Engagement in Rare Metabolic Disease Studies
