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    DRUG CLASS:

    Dystrophin expression stimulant

    Related drugs:
    26d
    DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov)
    P1/2, N=88, Recruiting, Dyne Therapeutics | Trial completion date: Nov 2026 --> Nov 2029 | Trial primary completion date: Nov 2026 --> Nov 2029
    Trial completion date • Trial primary completion date
    2ms
    ESSENCE: Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov)
    P3, N=228, Active, not recruiting, Sarepta Therapeutics, Inc. | Trial primary completion date: Oct 2025 --> Nov 2024
    Trial primary completion date
    2ms
    EXPLORE44OLE: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants with Mutations Amenable to Exon44 Skipping (clinicaltrials.gov)
    P2, N=35, Recruiting, Avidity Biosciences, Inc. | Enrolling by invitation --> Recruiting | N=24 --> 35
    Enrollment status • Enrollment change
    2ms
    EXPLORE44: Study of AOC 1044 in Healthy Adult Volunteers and Participants with Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (clinicaltrials.gov)
    P1/2, N=64, Active, not recruiting, Avidity Biosciences, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
    2ms
    EVOLVE: A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice (clinicaltrials.gov)
    P=N/A, N=300, Enrolling by invitation, Sarepta Therapeutics, Inc.
    New trial
    2ms
    A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P1, N=22, Active, not recruiting, Pfizer | Phase classification: P1b --> P1
    Phase classification • Gene therapy
    |
    CRP (C-reactive protein)
    2ms
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=43, Recruiting, Solid Biosciences Inc. | Trial completion date: May 2030 --> May 2031 | Trial primary completion date: May 2026 --> May 2027
    Trial completion date • Trial primary completion date • Gene therapy
    4ms
    GEN6050XIIT: A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (clinicaltrials.gov)
    P1, N=3, Recruiting, Peking Union Medical College Hospital | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    5ms
    IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov)
    P1/2, N=12, Active, not recruiting, Solid Biosciences Inc. | Trial completion date: Dec 2027 --> Oct 2026
    Trial completion date
    5ms
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=6, Recruiting, Solid Biosciences Inc. | Trial completion date: Jan 2030 --> May 2030 | Trial primary completion date: Jan 2026 --> May 2026
    Trial completion date • Trial primary completion date • Gene therapy
    7ms
    GEN6050XIIT: A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (clinicaltrials.gov)
    P1, N=3, Not yet recruiting, Peking Union Medical College Hospital
    New P1 trial • Gene therapy
    8ms
    Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P2, N=10, Active, not recruiting, Pfizer | Trial completion date: Jun 2028 --> Jan 2029 | Trial primary completion date: Jul 2024 --> Dec 2024
    Trial completion date • Trial primary completion date
    8ms
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=6, Recruiting, Solid Biosciences Inc. | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    9ms
    DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov)
    P1/2, N=88, Recruiting, Dyne Therapeutics | N=48 --> 88
    Enrollment change
    9ms
    A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P1/2, N=18, Recruiting, BioMarin Pharmaceutical
    New P1/2 trial
    9ms
    Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) (clinicaltrials.gov)
    P1/2, N=11, Active, not recruiting, Wave Life Sciences Ltd. | Recruiting --> Active, not recruiting | Phase classification: P1b/2a --> P1/2 | Trial completion date: Dec 2022 --> May 2025 | Trial primary completion date: Dec 2022 --> Jan 2025
    Enrollment closed • Phase classification • Trial completion date • Trial primary completion date
    9ms
    NS-050/NCNP-03 in Boys With DMD (Meteor50) (clinicaltrials.gov)
    P1/2, N=20, Not yet recruiting, NS Pharma, Inc. | Trial completion date: Jul 2026 --> May 2027 | Initiation date: Oct 2023 --> Jul 2024 | Trial primary completion date: Jun 2026 --> May 2027
    Trial completion date • Trial initiation date • Trial primary completion date
    9ms
    EXPLORE44OLE: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping (clinicaltrials.gov)
    P2, N=24, Enrolling by invitation, Avidity Biosciences, Inc.
    New P2 trial
    9ms
    Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (clinicaltrials.gov)
    P3, N=74, Active, not recruiting, NS Pharma, Inc. | Enrolling by invitation --> Active, not recruiting | Trial completion date: Jun 2026 --> Nov 2025 | Trial primary completion date: Jun 2026 --> Oct 2025
    Enrollment closed • Trial completion date • Trial primary completion date
    11ms
    NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov)
    P2, N=20, Recruiting, NS Pharma, Inc. | Not yet recruiting --> Recruiting | Trial completion date: May 2025 --> Sep 2025 | Initiation date: Aug 2023 --> Jan 2024 | Trial primary completion date: May 2025 --> Sep 2025
    Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date
    12ms
    MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov)
    P2, N=62, Active, not recruiting, Sarepta Therapeutics, Inc. | Trial completion date: Mar 2025 --> Jan 2029
    Trial completion date
    12ms
    A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) (clinicaltrials.gov)
    P3, N=160, Active, not recruiting, Sarepta Therapeutics, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
    1year
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=6, Not yet recruiting, Solid Biosciences Inc.
    New P1/2 trial • Gene therapy
    1year
    Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) (clinicaltrials.gov)
    P3, N=77, Completed, NS Pharma, Inc. | Recruiting --> Completed | Trial completion date: Dec 2024 --> Oct 2023 | Trial primary completion date: Nov 2024 --> Oct 2023
    Trial completion • Trial completion date • Trial primary completion date
    1year
    Two year muscle MRI observations from a phase 1b trial of fordadistrogene movaparvovec (PF‑06939926) for Duchenne muscular dystrophy (DMD) (ESGCT 2023)
    No abstract available
    P1 data
    1year
    A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. (clinicaltrials.gov)
    P2, N=3, Completed, Kevin Flanigan | Active, not recruiting --> Completed
    Trial completion
    2years
    Transformation from polycythemia vera to acute promyelocytic leukemia: Case report and literature review. (PubMed, Medicine (Baltimore))
    Polycythemia vera can transform into acute promyelocytic leukemia; therefore, it is important to review bone aspiration and other tests to perform a comprehensive assessment and monitor the disease status, to detect disease progression and intervene early when it transforms into acute promyelocytic leukemia.
    Review • Journal • Tumor Mutational Burden
    |
    TMB (Tumor Mutational Burden) • JAK2 (Janus kinase 2) • RARA (Retinoic Acid Receptor Alpha) • WT1 (WT1 Transcription Factor) • JAK3 (Janus Kinase 3)
    |
    JAK2 V617F • JAK2 mutation • WT1 mutation • JAK3 mutation
    |
    arsenic trioxide • hydroxyurea
    over2years
    Upregulation of Wilms' Tumor 1 in epicardial cells increases cardiac fibrosis in dystrophic mice. (PubMed, Cell Death Differ)
    The present study shows that Wilms' Tumor 1 (Wt1) upregulation in epicardial cells is primarily responsible for cardiac fibrosis and dysfunction of dystrophic mice and likely of DMD patients. Metformin effectively prevents cardiac fibrosis and improves cardiac function in dystrophic mice, thus representing a treatment option for DMD patients on top of existing therapies.
    Preclinical • Journal
    |
    WT1 (WT1 Transcription Factor)
    |
    metformin
    over4years
    [VIRTUAL] Retroperitoneal Lymphadenopathy as the First Manifestation of Lymphangioleiomyomatosis (LAM) in Elderly Female (ATS-I 2020)
    Given her lack of symptoms, there was no need to pursue treatment with sirolimus and the patient is being followed clinically with serial PFTs. VEGF-D level was borderline elevated at 753 pg/ml...In our case HRCT subsequently showed very few thin-walled pulmonary cysts. It is also highly unusual to be first diagnosed at this advanced age.
    Clinical
    |
    VEGFA (Vascular endothelial growth factor A)
    |
    sirolimus
    over4years
    Retroperitoneal Lymphadenopathy as the First Manifestation of Lymphangioleiomyomatosis (LAM) in Elderly Female (ATS 2020)
    Given her lack of symptoms, there was no need to pursue treatment with sirolimus and the patient is being followed clinically with serial PFTs. VEGF-D level was borderline elevated at 753 pg/ml.Discussion LAM is often diagnosed in women of child-bearing age and frequently manifests with pulmonary symptoms...In our case HRCT subsequently showed very few thin-walled pulmonary cysts. It is also highly unusual to be first diagnosed at this advanced age.
    Clinical
    |
    VEGFA (Vascular endothelial growth factor A)
    |
    sirolimus