Concepedia

Risdiplam Therapy

Risdiplam Therapy is a pharmacotherapeutic approach that belongs to the class of survival motor neuron-2 (SMN2) splicing modulators, distinguished by its administration of the small molecule risdiplam to enhance full-length SMN protein production in patients with spinal muscular atrophy.

42

Publications

2.7K

Citations

577

Authors

168

Institutions

Publications per year

2017–2026

42

Parents

Publications

42

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( <i>SMN2</i> ) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Hasane Ratni, Martin Ebeling, John D. Baird et al. · Journal of Medicinal Chemistry · 2018 · 520 citations · Full text

, , +14

Risdiplam in Type 1 Spinal Muscular Atrophy

Giovanni Baranello, Basil T. Darras, Nicolas Deconinck et al. · New England Journal of Medicine · 2021 · 419 citations · Full text

, , +13

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Basil T. Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska et al. · New England Journal of Medicine · 2021 · 274 citations · Full text

, , +17

Risdiplam distributes and increases <scp>SMN</scp> protein in both the central nervous system and peripheral organs

Agnès Poirier, Marla Weetall, Katja Heinig et al. · Pharmacology Research & Perspectives · 2018 · 169 citations · Full text

, , +15

Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines

Hasane Ratni, R. Scalco, Alexander Stephan · ACS Medicinal Chemistry Letters · 2021 · 134 citations · Full text

, , +7

1–5 of 42

Authors

577

  1. KG
    Ksenija Gorni

    Roche (Switzerland), Switzerland

    16 publications · 1.3K citations

    13

    h-index

  2. HK
    Heidemarie Kletzl

    Roche (Switzerland), Switzerland

    14 publications · 1.1K citations

    12

    h-index

  3. RS
    R. Scalco

    University College London, United Kingdom

    12 publications · 1.3K citations

    12

    h-index

  4. GB
    Giovanni Baranello

    Fondazione IRCCS Istituto Neurologico Carlo Besta, Italy

    12 publications · 1.1K citations

    11

    h-index

  5. EM
    Eugenio Mercuri

    Università Cattolica del Sacro Cuore, Italy

    12 publications · 925 citations

    11

    h-index

1–5 of 577

Institutions

168

  1. Roche (Switzerland)

    Basel, Switzerland

    18 h-index

    152 publications

    19.5K citations

  2. Roche (United Kingdom)

    Welwyn Garden City, United Kingdom

    12 h-index

    23 publications

    1.9K citations

  3. University College London

    London, United Kingdom

    11 h-index

    47 publications

    12.5K citations

  4. Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Italy

    10 h-index

    18 publications

    2.1K citations

  5. Centro Clinico Nemo

    Milan, Italy

    10 h-index

    19 publications

    1.3K citations

  6. Great Ormond Street Hospital

    London, United Kingdom

    9 h-index

    28 publications

    7.6K citations

  7. Stanford University

    Stanford, United States

    8 h-index

    9 publications

    867 citations

  8. Hôpital Armand-Trousseau

    Paris, France

    7 h-index

    8 publications

    1.1K citations

1–8 of 168

Venues

  1. Neurology

    5 publications · 96 citations

    5

    h-index

  2. Muscle & Nerve

    4 publications · 129 citations

    4

    h-index

  3. Annals of Clinical and Translational Neurology

    ACTN · 2 publications · 74 citations

    2

    h-index

  4. Journal of Neurology

    2 publications · 120 citations

    2

    h-index

  5. Nucleic Acids Research

    2 publications · 71 citations

    2

    h-index

1–5