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
- Learn the pathogenic mechanisms of some important neurogenetic and neurometabolic diseases in children.
- Gain knowledge about recent advances in novel treatments for some important neurogenetic and neurometabolic diseases.
Impact Statements
- The recognition of clinical features of certain rare neurogenetic and neurometabolic diseases.
- 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
