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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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Press Releases
Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan...
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Satellos submits IND to FDA for Phase 2 SAT-3247 Clinical Trial in Duchenne Muscular Dystrophy
Satellos Bioscience announced the submission of an Investigational New Drug (IND) application to the...
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FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy
Nippon Shinyaku announced that the U.S. Food and Drug Administration (FDA) has granted Orphan...
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Italfarmaco and Multicare Pharma Sign Exclusive Distribution Deal for Givinostat (Duvyzat), a DMD Treatment in Brazil
Italfarmaco announced an exclusive agreement with Multicare Pharma, a prominent pharmaceutical company in Latin...
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Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial
Avidity Biosciences announced positive new data from participants treated continuously with del-zota for one...
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Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy
Belief BioMed, an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced...
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Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting
Keros Therapeutics announced that it presented additional clinical data from its Phase 1 clinical...
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SQY Therapeutics Receives Orphan Drug Designation from the European Medicines Agency for Exon 51 Skipping Therapy SQY51 for Treatment of Duchenne Muscular Dystrophy
SQY51 had already obtained Orphan Drug Designation from the US Food and Drug Administration...
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Santhera Enters into Agreement with Ikris Pharma Network for the Distribution of AGAMREE (Vamorolone) in India
Santhera Pharmaceuticals announces the signing of an exclusive agreement with Ikris Pharma Network (Ikris)...
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ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy
ITF Therapeutics affiliate of Italfarmaco, today announced publication of positive long-term safety and efficacy...
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Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy
Keros Therapeutics today announced the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug...
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Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026
Santhera Pharmaceuticals announces the signing of an exclusive agreement with Uniphar to manage the...
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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...