Neurometabolic disorders: Novel Advanced Treatment in Neurogenetic Disorders

Course Description: The aim of this symposium is to present novel treatments for neurogenetic disorders, which will enhance our understanding of the future potential for treatment of a range of neurogenetic conditions.

Learning Objectives

1. Learn the pathogenic mechanisms of some important neurogenetic and neurometabolic diseases in children.
2. Gain knowledge about recent advances in novel treatments for some important neurogenetic and neurometabolic diseases.

Impact Statements

1. The recognition of clinical features of certain rare neurogenetic and neurometabolic diseases.
2. Knowledge about recent advances in the treatment of neurogenetic and neurometabolic diseases.

Organizer: Wang-Tso Lee, MD, PhD
National Taiwan University Children’s Hospital, Taipei, Taiwan

Recent Advances in SSADH Deficiency, a Disorder of GABA Metabolism
Phillip L. Pearl, MD; Boston Children’s Hospital, Harvard Medical School, Boston, Massachusetts, USA

Recent Advance in Possible Probiotic Treatment for Neurogenetic Disorders
Wang-Tso Lee, MD, PhD

Gene Therapy for AADC Deficiency and other Neurogenetic Disorders
Toni Pearson, MBBS; Washington University School of Medicine, St. Louis, Missouri, USA

Recent Advance in Targeted Treatment in Children with NCLs
Nicola Specchio, MD, PhD
Bambino Gesu’ Children’s Hospital, IRCCS, Rome, Italy