Research
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 muscular dystrophy (Duchenne) that uses exon-skipping technology and RNA-based medicines, including antisense oligonucleotides (ASOs). The company presented the new findings at the 22nd Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2026. The results showed...
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STM-01 Enters Phase 2 Clinical Trial for Duchenne Muscular Dystrophy
Secretome Therapeutics has announced the start of THRIVE-2, a Phase 2 clinical study evaluating its investigational therapy STM-01 in people living with Duchenne muscular dystrophy (DMD). The study is now open for enrollment, with the first participant expected to enroll soon.What Is THRIVE-2?THRIVE-2 is designed as a multicenter, multi-dose,...
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Research
Researchers in Madrid Make Important Strides in the Cure of Duchenne Muscular Dystrophy with Nanoparticles
The Community of Madrid has achieved significant progress in therapy for Duchenne muscular dystrophy,...
Research
Metagenomi’s New CRISPR System (VK CAST) Enables Large Gene Integration
Metagenomi, a precision genetic medicines company committed to developing curative therapeutics for patients using...
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Hydrogel Delivery System Holds Potential for Cell-Based Therapy of Duchenne Muscular Dystrophy
In a mouse model of muscular dystrophy, researchers in the UK announced a clinically...
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OligomicsTx Gene Therapy Study Shows Promise in Treating Duchenne Muscular Dystrophy
The University of Alberta researchers develop a molecule that bypasses genetic mutation to restore...
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Stitch RNA: New Method for Duchenne Gene Therapies
The Stitch RNA (StitchR) method could accelerate the development of new drugs that could...
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AlltRNA’s Engineered tRNA Technology Could Offer Hope for Nonsense Mutations
Regardless of the impacted protein, Alltrna is creating designed tRNA medications that can read...
Research
Nanoparticles Used as Vehicle to Deliver MicroRNAs to Cells and Treat Duchenne Muscular Dystrophy
In a collaboration between the IMDEA Nanociencia Institute (Madrid), Università Cattolica del Sacro Cuore...
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ENCell Confirms Safety of DMD Stem Cell Therapy EN001 in Phase 1 Clinical Trial in Korea
The bio corporation ENCell reported on February 13 that it confirmed the safety and...
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Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers
Satellos Bioscience, a public biotech company developing new small molecule therapeutic approaches to improve...
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Sarcomatrix to Present its Latest Advancements in the Development of its Lead Drug Candidate, S-969
Sarcomatrix Therapeutics, a clinical-stage biotechnology company focused on revolutionizing treatments for muscle-related diseases, today...
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Upcoming Exon 44 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
Exon 44 skipping therapies represent a promising area in the treatment of Duchenne Muscular...
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Russian Biotech Company Generium has Begun Clinical Trials of a Gene Therapy for Duchenne Muscular Dystrophy
Clinical studies for a therapy for Duchenne muscular dystrophy have begun at Sirius University...
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DMD
What Is Duchenne Muscular Dystrophy (DMD)?: 108 FAQs
Duchenne muscular dystrophy (DMD), commonly known as Duchenne, is a rare genetic disorder that...
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,...
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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...
Research
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...
Press Releases
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...
Press Releases
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...