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Research article

First published 2018-2-21

Mutation Spectrum of GNE Myopathy in the Indian Sub-Continent

Sudha Bhattacharya, Satish V. Khadilkar, Atchayaram Nalini [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024

Induced Muscle and Liver Absence of Gne in Postnatal Mice Does Not Result in Structural or Functional Muscle Impairment

Avi Harazi, Lena Yakovlev, Nili Ilouz [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2022-01-04

Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy

Stella Mitrani-Rosenbaum, Lena Yakovlev, Michal Becker Cohen [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2020

Upregulation of Hallmark Muscle Genes Protects Gne M743T/M743T Mutated Knock-In Mice From Kidney and Muscle Phenotype

Hadar Benyamini, Yehuda Kling, Lena Yakovlev [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2016

GNE-Myopathy in a Greek Romani Family with Unusual Calf Phenotype and Protein Aggregation Pathology

G.K. Papadimas, A. Evilä, C. Papadopoulos [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-09-08

Development of Assays to Measure GNE Gene Potency and Gene Replacement in Skeletal Muscle

Deborah A. Zygmunt, Patricia Lam, Anna Ashbrook [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2021-07-30

Thigh and Leg Muscle MRI Findings in GNE Myopathy

Farzad Fatehi, Soroor Advani, Ali Asghar Okhovat [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2009

Safety and in vivo Expression of a GNE-Transgene: A Novel Treatment Approach for Hereditary Inclusion Body Myopathy-2

Anagha P. Phadke, Chris Jay, Salina J. Chen [...]

Gene Regulation and Systems Biology

Open access

Research article

First published 2023-07-04

Phase II/III Study of Aceneuramic Acid Administration for GNE Myopathy in Japan

Naoki Suzuki, Madoka Mori-Yoshimura, Masahisa Katsuno [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2025-3

Ultra-Orphan drug development for GNE Myopathy: A synthetic literature review and meta-analysis

Naoki Suzuki, Madoka Mori-Yoshimura, Ichizo Nishino [...]

Journal of Neuromuscular Diseases

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