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-Reference-Cited by-同舟云学术

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

Published:2016-03-03 Issue:1 Volume:3 Page:49-66
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

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

Cited by 35 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Safety and efficacy of aceneuramic acid in GNE myopathy: open-label extension study;Journal of Neurology, Neurosurgery & Psychiatry;2024-06-05

2. Large phenotypic diversity by genotype in patients with GNE myopathy: 10 years after the establishment of a national registry in Japan;Journal of Neurology;2024-05-01

3. Pharmacokinetics and clinical efficacy of 6′-sialyllactose in patients with GNE myopathy: Randomized pilot trial;Biomedicine & Pharmacotherapy;2023-12

4. Recent advances on N-acetylneuraminic acid: Physiological roles, applications, and biosynthesis;Synthetic and Systems Biotechnology;2023-09

5. Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan;Orphanet Journal of Rare Diseases;2023-08-11