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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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Aji Bio-Pharma and Gene Tools LLC Announce Collaboration for Duchenne Muscular Dystrophy (DMD)
Aji Bio-Pharma a leading provider of biopharmaceutical contract development and manufacturing services, and Gene...
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Satellos Announces First Adult Patient Dosed in LT-001, an Open-Label, Long-Term Follow-Up Study of SAT-3247 in Duchenne Muscular Dystrophy
Satellos Bioscience, announced that the first patient has been dosed in the open-label, long-term...
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Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping
Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an...
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Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026
Avidity Biosciences announced that the Company completed a positive pre-BLA meeting with the U.S....
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Satellos Announces New Data Further Demonstrating Safety, Tolerability, and Functional Impact of SAT-3247 in First-in-Human Trial of Adults with Duchenne Muscular Dystrophy
Satellos Bioscience announced new data further demonstrating tolerability and initial efficacy of SAT-3247 in...
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Genethon Confirms 2-Year Efficacy in Duchenne Muscular Dystrophy Patients Treated With Its Low Dose Micro-Dystrophin Gene Therapy (GNT0004)
Genethon, released positive long-term efficacy data revealing three Duchene muscular dystrophy patients showed significant...
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Santhera Secures Agreement with Biomedica for the Distribution of AGAMREE (Vamorolone) in Russia
Santhera announces the signing of an exclusive agreement with Biomedica for the distribution of...
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SonoThera Presents New Data at ESGCT 2025 Demonstrating Expression of Full-Length Human Dystrophin Using Targeted, Ultrasound-Mediated Delivery to Treat Duchenne Muscular Dystrophy
SonoThera, a biotechnology company dedicated to treating the root cause of human diseases by...
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New Data Presented on Duvyzat (Givinostat) for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress
Italfarmaco announced that additional data on givinostat for the treatment of Duchenne muscular dystrophy...
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Form Bio and Cure Rare Disease Partner to Accelerate Genetic Medicine Development, Advancing CRD’s Duchenne Muscular Dystrophy Program
Form Bio, a leader in AI-powered genetic medicine development and genome engineering, announced a...
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Health Canada Approves AGAMREE (vamorolone) as the First Treatment for Duchenne Muscular Dystrophy in Canada
Kye Pharmaceuticals announced that Health Canada has approved AGAMREE (vamorolone) for the treatment of...
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Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy
Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length...
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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...