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    DRUG CLASS:

    Dystrophin expression stimulant

    Related drugs:
    15d
    Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) (clinicaltrials.gov)
    P3, N=90, Recruiting, Dyne Therapeutics
    New P3 trial
    15d
    SGT-003-301: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (2025-522949-22-00)
    P2/3, N=20, Not yet recruiting, Solid Biosciences Inc.
    New P2/3 trial
    22d
    AOC 1044-CS3: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping (2025-523087-20-00)
    P2/3, N=26, Not yet recruiting, Avidity Biosciences Inc.
    New P2/3 trial
    28d
    SAFARI44: A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping (clinicaltrials.gov)
    P3, N=70, Not yet recruiting, Avidity Biosciences, Inc.
    New P3 trial
    29d
    Study of Pembrolizumab combined with Ataluren in Patients with metastatic pMMR and dMMR colorectal cancer adenocarcinomas or metastatic dMMR endometrial carcinoma: the ATAPEMBRO study (2023-509457-31-00)
    P1/2, N=47, Completed, Amsterdam UMC | Active, not recruiting --> Completed
    Trial completion • dMMR • pMMR
    |
    Keytruda (pembrolizumab) • Translarna (ataluren)
    1m
    An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P2, N=18, Enrolling by invitation, BioMarin Pharmaceutical
    New P2 trial
    1m
    Ion-Channel-Mediated Drug Repurposing Opportunities Validated by Single-Cell Perturbation in Colorectal Cancer. (PubMed, Int J Mol Sci)
    Immune checkpoint receptors (LAG3, CD27) connect via PPI intermediates to Ca2+ and K+ channels, targetable by relatlimab (FDA-approved) and varlilumab (Phase 2). This work maps previously unknown links between CRC driver genes and ion channel regulation, with the ataluren-RPS21-KCNQ2 axis ready for pharmacological testing.
    Journal • IO biomarker
    |
    LAG3 (Lymphocyte Activating 3) • CD27 (CD27 Molecule)
    |
    relatlimab (BMS-986016) • varlilumab (CDX 1127) • Translarna (ataluren)
    2ms
    NS-050/NCNP-03 in Boys With DMD (Meteor50) (clinicaltrials.gov)
    P1/2, N=20, Active, not recruiting, NS Pharma, Inc. | Recruiting --> Active, not recruiting | Trial completion date: May 2027 --> Mar 2028
    Enrollment closed • Trial completion date • First-in-human
    2ms
    Rescuing TP53 from nonsense: novel triazoles for translational readthrough via optimized drug design. (PubMed, Sci Rep)
    All four compounds successfully rescued p53 expression in H1299 R213X cells, outperforming Ataluren and matching G418 at significantly lower concentrations. The restored p53 exhibited nuclear localization upon genotoxic stress and induced transcription of canonical targets. These findings highlight the therapeutic potential of these compounds for treating TP53 nonsense mutations in cancer and lay the groundwork for the development of targeted nonsense mutation-specific treatment for a wide range of pathologies, including new emergent p53 related diseases.
    Journal
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    Translarna (ataluren)
    3ms
    351-202: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants with Duchenne Muscular Dystrophy (2024-511656-41-00)
    P1/2, N=3, Not yet recruiting, Biomarin Pharmaceutical Inc.
    New P1/2 trial
    3ms
    Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) (clinicaltrials.gov)
    P3, N=270, Completed, PTC Therapeutics | Enrolling by invitation --> Completed | Trial completion date: Apr 2025 --> Feb 2026 | Trial primary completion date: Apr 2025 --> Feb 2026
    Trial completion • Trial completion date • Trial primary completion date
    |
    Translarna (ataluren)
    3ms
    Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P2, N=175, Enrolling by invitation, Wave Life Sciences Ltd. | Not yet recruiting --> Enrolling by invitation
    Enrollment open