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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Researchers at The University of Minnesota Find a New Way to Treat Duchenne Muscular Dystrophy: Bottlebrush Polymer
A novel strategy has been discovered by researchers at the University of Minnesota Medical...
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WVE-N531 Exon Skipping Therapy Phase 2 Clinical Trial Begins for Duchenne Muscular Dystrophy
Wave Life Sciences Ltd. announced on its ClinicalTrials.gov website that a phase 2 clinical...
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SPOT-mRNA03 Gene Therapy for Duchenne Muscular Dystrophy Begins Early Phase 1 Clinical Trial
The early phase 1 study (NCT07188012) of SPOT-mRNA03 gene therapy for Duchenne Muscular Dystrophy...
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Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males
The phase 3 study (NCT07160634) of the SGT-003 gene therapy developed by Solid Biosciences...
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GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium
Generium is a Russian innovative biotechnology company has received approval to conduct a clinical...
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ITF Therapeutics Launching A Study to Evaluate the Real-World Experience of Duvyzat (Givinostat) in Patients With Duchenne Muscular Dystrophy
The purpose of this prospective observational study (NCT07127978) is to assess the safety, tolerability,...
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Revidia Therapeutics MSI-1436 Holds Promise for Treating Heart Failure in DMD Patients
Human babies may indeed repair myocardial damage and fully recover normal cardiac function following...
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Preclinical Study: Cell Therapy Prevents Damage From Duchenne Muscular Dystrophy
A cell therapy preserves muscle structure and function in laboratory mice with a type...
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Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Genethon has received approvals from regulatory authorities, MHRA and EMA, to begin pivotal Phase...
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UW Medicine Announces New Gene Therapy Research for Duchenne Muscular Dystrophy
For those who suffer from Duchenne muscular dystrophy (DMD), a novel gene therapy holds...
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INS1201 Gene Therapy Phase 1 Clinical Trial Begins
Insmed was announced on the page published on ClinicalTrials.gov with the ID NCT06817382 that...
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Medera and University of Kansas Medical Center Announce First Patient Treated in Pioneering Gene Therapy Trial (SRD-001) for Duchenne Muscular Dystrophy Associated Cardiomyopathy
Medera a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics,...
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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,...
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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...
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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...