Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study

Author:

Argov Zohar1,Caraco Yoseph1,Lau Heather2,Pestronk Alan3,Shieh Perry B.4,Skrinar Alison5,Koutsoukos Tony5,Ahmed Ruhi5,Martinisi Julia5,Kakkis Emil5

Affiliation:

1. Hadassah-Hebrew University Medical Center, Jerusalem, Israel

2. New York University School of Medicine, New York, NY, USA

3. Washington University Medical Center, St. Louis, MO, USA

4. University of California Los Angeles Medical Center, Los Angeles, CA, USA

5. Ultragenyx Pharmaceutical, Novato, CA, USA

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

Reference22 articles.

1. GNE myopathy: Current update and future therapy;Nishino;Journal of Neurology, Neurosurgery, and Psychiatry,2015

2. “Rimmed vacuole myopathy” sparing the quadriceps: A unique disorder in iranian jews;Argov;J Neurol Sci,1984

3. Hereditary inclusion body myopathy: A decade of progress;Huizing;Biochim Biophys Acta,2009

4. Hereditary inclusion body myopathy and other rimmed vacuolar myopathies;Argov;Handbook of Clinical Neurology,2007

5. Distal myopathy with rimmed vacuole formation;Sunohara;A follow-up study. Brain: A Journal of Neurology,1989

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