Concepedia

Substrate Reduction Therapy

Substrate Reduction Therapy is a pharmacological strategy that belongs to the class of therapeutic interventions for lysosomal storage disorders, distinguished by its approach of decreasing the production of accumulated substrates to restore metabolic equilibrium.

136

Publications

6.4K

Citations

858

Authors

300

Institutions

Publications per year

2017–2026

62

Parents

Publications

136

Chemical chaperone therapy for brain pathology in G <sub>M1</sub> -gangliosidosis

Junichiro Matsuda, Osamu Suzuki, Akihiro Oshima et al. · Proceedings of the National Academy of Sciences · 2003 · 264 citations · Full text

, , +13

Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1

Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia et al. · JAMA · 2015 · 156 citations

, , +11

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

Elena Lukina, Nora Watman, Elsa Ávila Arreguin et al. · Blood · 2010 · 154 citations · Full text

, , +3

Recent advances in the diagnosis and management of Gaucher disease

Sam E. Gary, Emory Ryan, Alta Steward et al. · Expert Review of Endocrinology & Metabolism · 2018 · 140 citations · Full text

, , +17

Genistein Improves Neuropathology and Corrects Behaviour in a Mouse Model of Neurodegenerative Metabolic Disease

Marcelina Malinowska, Fiona L. Wilkinson, Kia Langford‐Smith et al. · PLoS ONE · 2010 · 138 citations · Full text

, , +15

1–5 of 136

Authors

858

  1. MP
    Michel Peterschmitt

    Centre de Coopération Internationale en Recherche Agronomique pour le Développement, France

    11 publications · 761 citations

    11

    h-index

  2. SH
    Seng H. Cheng

    Framingham State University, United States

    10 publications · 593 citations

    10

    h-index

  3. AZ
    Ari Zimran

    Shaare Zedek Medical Center, Israel

    9 publications · 322 citations

    9

    h-index

  4. ÖG
    Özlem Göker-Alpan

    Lysosomal and Rare Disorders Research and Treatment Center, United States

    7 publications · 169 citations

    7

    h-index

  5. SJ
    Sebastiaan J.M. Gaemers

    Sanofi (United States), United States

    7 publications · 359 citations

    7

    h-index

1–5 of 858

Institutions

300

  1. Sanofi (United States)

    Bridgewater, United States

    12 h-index

    69 publications

    2.4K citations

  2. University College London

    London, United Kingdom

    11 h-index

    12 publications

    691 citations

  3. Shaare Zedek Medical Center

    Jerusalem, Israel

    11 h-index

    28 publications

    966 citations

  4. New York University

    New York, United States

    9 h-index

    10 publications

    762 citations

  5. Icahn School of Medicine at Mount Sinai

    New York, United States

    7 h-index

    18 publications

    722 citations

  6. University of Kansas Medical Center

    Kansas City, United States

    6 h-index

    13 publications

    690 citations

  7. University of Gdańsk

    Gdansk, Poland

    6 h-index

    24 publications

    1.8K citations

  8. University of Oxford

    Oxford, United Kingdom

    6 h-index

    30 publications

    2.7K citations

1–8 of 300

Venues

  1. Molecular Therapy

    8 publications · 544 citations

    8

    h-index

  2. Orphanet Journal of Rare Diseases

    OJRD · 7 publications · 261 citations

    7

    h-index

  3. Journal of Inherited Metabolic Disease

    JIMD · 6 publications · 307 citations

    6

    h-index

  4. Molecular Genetics and Metabolism Reports

    5 publications · 94 citations

    5

    h-index

  5. JIMD Reports

    Book series · 5 publications · 177 citations

    5

    h-index

1–5