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AFM Telethon

Sponsors » ULTRAGENYX

Lunch symposium sponsored by Ultragenyx

GNE Myopathy Symposium

Thursday, 17 March 2016
12:30-14:00 hrs
Room Pasteur


Welcome Address & Introduction

Dr. Anthony Behin, PH

Neurologue, médecin agréé
Centre de Référence de Pathologie Neuromusculaire Paris-Est
Institut de Myologie
GH Pitié-Salpêtrière, Paris, France


GNE Myopathy Disease Overview

Zohar Argov, MD
Professor (Emeritus) of Neurology,
Hadassah-Hebrew University Medical School, Jerusalem, Israel


Natural History of GNEM

Hanns Lochmüller, MD, FAAN
Chair of Experimental Myology
Deputy Director, Institute of Genetic Medicine
The John Walton Muscular Dystrophy Research Centre
MRC Centre for Neuromuscular Diseases
Newcastle University, UK


Aceneuramic Acid (sialic acid) Extended Release (ER) for the Treatment of GNE Myopathy

Edward Conner, MD
Executive Medical Director, Clinical Sciences
Ultragenyx Pharmaceutical Inc.

 

Description:

Review of the Ace-ER clinical program including: safety and efficacy of Ace-ER, Phase I Pharmacokinetic Profile and Safety Study, Phase II Dosing Study, and current Phase III Study Protocol.