Inborn Errors of Metabolism Drug Development Summit


The Inborn Errors of Metabolism Drug Development Summit is a conference focused on key patient access, regulatory, clinical and commercial aspects of gene therapy.

The Inborn Errors of Metabolism Drug Development Summit covers topics such as:

  • 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

Future Events

Past Events


Please, check the official conference website for possible changes, before you make any traveling arrangements

Prices are for evaluation only.

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