^
    Contact us  to learn more about
    our Premium Content:  News alerts, weekly reports and conference planners
    DRUG CLASS:

    Dystrophin expression stimulant

    Related drugs:
    5d
    A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) (clinicaltrials.gov)
    P3, N=80, Recruiting, Solid Biosciences Inc. | Not yet recruiting --> Recruiting
    Enrollment open
    8d
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=60, Recruiting, Solid Biosciences Inc. | N=40 --> 60
    Enrollment change
    22d
    Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping (clinicaltrials.gov)
    P=N/A, N=0, Available, Avidity Biosciences, Inc.
    New trial
    29d
    Ataluren-Induced Functional Restoration of Neurofibromin in Fibroblasts From Neurofibromatosis Type 1 Patients With Nonsense Mutations. (PubMed, MedComm (2020))
    Interestingly, AMPD3 can be an effective therapeutic target for NF1-associated diseases. Together, our study suggests that ataluren can be considered a therapeutic agent for some NF1NS/+ patients and contributes to expanding insights into NF1 therapy.
    Journal
    |
    NF1 (Neurofibromin 1)
    |
    Translarna (ataluren)
    29d
    ESSENCE: Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov)
    P3, N=228, Completed, Sarepta Therapeutics, Inc. | Active, not recruiting --> Completed
    Trial completion
    2ms
    Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P2, N=10, Terminated, Pfizer | Active, not recruiting --> Terminated; All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003
    Trial termination
    2ms
    A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. (clinicaltrials.gov)
    P3, N=7, Terminated, Pfizer | Active, not recruiting --> Terminated; All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
    Trial termination
    2ms
    TREM2-Mediated Cholesterol Efflux in Macrophages Inhibits Anti-Tumor Immunity via Limitation of CD4+ T and NK Cells. (PubMed, Adv Sci (Weinh))
    After screening food and drug administration (FDA)-approved drugs, bortezomib and ataluren are found to effectively inhibit TREM2 expression in TAMs, indicating a potential therapeutic strategy against TREM2. This study elucidates the mechanism by which TREM2 shapes the immunosuppressive microenvironment and promotes tumorigenesis, highlighting TREM2 as a target for cancer immunotherapy.
    Journal • IO biomarker
    |
    CD4 (CD4 Molecule) • CX3CL1 (C-X3-C Motif Chemokine Ligand 1) • ABCA1 (ATP Binding Cassette Subfamily A Member 1) • TREM2 (Triggering Receptor Expressed On Myeloid Cells 2)
    |
    bortezomib • Translarna (ataluren)
    2ms
    Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P2, N=175, Not yet recruiting, Wave Life Sciences Ltd.
    New P2 trial
    2ms
    Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) (clinicaltrials.gov)
    P1/2, N=26, Recruiting, Wave Life Sciences Ltd. | Active, not recruiting --> Recruiting
    Enrollment open
    3ms
    Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) (clinicaltrials.gov)
    P1/2, N=26, Active, not recruiting, Wave Life Sciences Ltd. | N=11 --> 26 | Trial completion date: Oct 2026 --> Apr 2027 | Trial primary completion date: Jan 2025 --> Jun 2026
    Enrollment change • Trial completion date • Trial primary completion date
    3ms
    A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov)
    P1, N=23, Terminated, Pfizer | Active, not recruiting --> Terminated; All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
    Trial termination