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Entrada Therapeutics ENTR-601-45: Early Exon 45 Skipping Study Shows Increased Dystrophin Production
Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201, evaluating ENTR-601-45 in people with Duchenne muscular dystrophy (Duchenne). The first group of participants showed a favorable safety profile and a small increase in dystrophin production after receiving the initial dose. The findings are an...
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Entrada Therapeutics Advances ENTR-601-44 to 18 mg/kg in Its Duchenne Exon 44 Skipping Study
Entrada Therapeutics has received clearance to test the highest dose in its Exon 44 Skipping Study for Duchenne muscular dystrophy (DMD). The independent Data Monitoring Committee (DMC) has recommended starting Cohort 3 at 18 mg/kg in the ELEVATE-44-201 clinical trial, following its review of available safety and pharmacokinetic data...
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IPS HEART Aims to Restore Full-Length Dystrophin with Two Regenerative Cell Therapies for Duchenne Muscular Dystrophy
IPS HEART has announced another important regulatory milestone after receiving FDA Rare Pediatric Disease...
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GEN6050X Exon 50 Skipping Base Editing Drug: One-Year Results Show Encouraging Progress for Duchenne Muscular Dystrophy
The GEN6050X exon 50 skipping base editing drug, developed by GenAssist Therapeutics, is attracting...
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Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen, an Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy (DMD)
Families affected by Duchenne muscular dystrophy (DMD) received encouraging news as Dyne Therapeutics announced...
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Santhera Expands Biomedica Agreement to Extend AGAMREE (Vamorolone) Distribution Across Central Asia and the Caucasus
Santhera today announces an expansion of its exclusive distribution agreement with Biomedica for AGAMREE...
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SAT-3247 Six-Month TRAILHEAD Results Show Reduced Muscle Fat and Lower CK in Adults with Duchenne muscular dystrophy
New six-month interim results from the TRAILHEAD clinical trial suggest that SAT-3247 may help...
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Satellos Receives FDA Fast Track Designation for SAT-3247 in Duchenne Muscular Dystrophy
Satellos Bioscience has announced that the U.S. Food and Drug Administration (FDA) has granted...
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Elixirgen and Nippon Shinyaku Partner on Full-Length Dystrophin Therapy for Duchenne
Elixirgen Therapeutics has announced a strategic option agreement with Nippon Shinyaku for the development...
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REGENXBIO Completes RGX-202 Confirmatory Study for Duchenne Muscular Dystrophy
REGENXBIO has announced the completion of patient dosing in the confirmatory study of RGX-202,...
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GEn1E Lifesciences and GEn-1123: A New Precision Medicine Approach for Duchenne Muscular Dystrophy (DMD)
The U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD)...
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RGX-202 Duchenne Gene Therapy Shows NSAA Improvement and Microdystrophin Expression — But Important Biomarker Data Missing
REGENXBIO recently announced positive topline results from the Phase 3 portion of the AFFINITY...
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Entrada Therapeutics Announces Phase 1/2 Results for ENTR-601-44 in Duchenne Muscular Dystrophy
Entrada Therapeutics announced early clinical trial results for ENTR-601-44, an experimental treatment for people...
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European Commission Grants Orphan Drug Designation to SGT-003 for Duchenne Muscular Dystrophy
The European Commission has granted Orphan Drug Designation to SGT-003, a promising gene therapy...
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Press Releases
Entrada Therapeutics ENTR-601-45: Early Exon 45 Skipping Study Shows Increased Dystrophin Production
Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201,...
Press Releases
Entrada Therapeutics Advances ENTR-601-44 to 18 mg/kg in Its Duchenne Exon 44 Skipping Study
Entrada Therapeutics has received clearance to test the highest dose in its Exon 44...
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Sapreme Therapeutics Reports Strong Preclinical Dystrophin Restoration With SPM-2551 in Duchenne
Sapreme Therapeutics has announced promising preclinical results for SPM-2551, an experimental treatment for Duchenne...
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Deramiocel Shows Positive 24-Month HOPE-3 Results in Duchenne
Capricor Therapeutics has reported new results from the HOPE-3 study evaluating Deramiocel in people...
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Ractigen Therapeutics Reports Positive First-in-Human Data for RAG-18 in Duchenne
Ractigen Therapeutics has presented positive first-in-human data for RAG-18 (NCT07282652), an investigational treatment for...
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Entrada Therapeutics’ ENTR-601-44 Shows Dose-Dependent Exon 44 Skipping in Early PK/PD Data
Entrada Therapeutics’ ENTR-601-44 is an investigational therapy designed for patients with Duchenne muscular dystrophy...
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NS Pharma’s Brogidirsen Shows 5-Year Clinical Data in DMD Patients Amenable to Exon 44 Skipping
NS Pharma’s brogidirsen (NS-089/NCNP-02) is an investigational therapy for Duchenne muscular dystrophy (DMD) designed...
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ELEVIDYS WMS 2026 Data: What They Show—and What They Don’t
Sarepta Therapeutics has presented new data on ELEVIDYS (delandistrogene moxeparvovec) at the 2026 World...