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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 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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Medera’s Gene Therapy for DMD Cardiomyopathy Gains Momentum with FDA Fast Track Designation
A promising advancement in gene therapy for DMD cardiomyopathy has emerged as Medera Inc....
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SGT-003 Gene Therapy: INSPIRE DUCHENNE Phase 3 Study Begins
Solid Biosciences has reported encouraging interim results from its ongoing Phase 1/2 INSPIRE DUCHENNE...
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Atossa Presented New (Z)-Endoxifen Research Showing Improved Muscle Function in Duchenne Mouse Model
At the 2026 MDA Clinical & Scientific Conference in Orlando, researchers from Atossa Therapeutics...
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Del-Zota Phase 3 SAFARI44 Clinical Trial for Duchenne Muscular Dystrophy: Study Design, Data, and Clinical Significance
The del-zota phase 3 SAFARI44 clinical trial for Duchenne muscular dystrophy represents a major...
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Satellos Announced the Start of Phase 2A (BASECAMP) Study of SAT-3247 in Ambulatory Patients with Duchenne
Satellos announced the commencement of a Phase 2A trial of SAT-3247 (NCT07287189) in ambulatory...
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China-Based Ractigen Therapeutics is Initiating Clinical Trials to Investigate The Safety and Tolerability of RAG-18 in Patients with DMD
According to Ractigen Therapeutics, this study is an open-label, single-arm, dose-escalation study designed to...
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Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy
Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the...
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Del-Zota Clinical Trial Begins in the US in Participants with DMD Mutations Amenable to Exon 44 Skipping
Avidity Biosciences announced the commencement of clinical trials to provide access to delpacibart...
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Researchers Develop Optimized Smad7 Gene Therapy to Target Duchenne Muscular Dystrophy
On November 25, 2025, researchers Buel D. Rodgers and Christopher W. Ward published a...
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Ali Taghizadeh Announces DMD ANKA mRNA Therapy, Currently in Clinical Trial in Turkiye
Turkish pharmacologist Assoc. Prof. Dr. Ali Taghizadeh announced in English to his followers abroad...
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Lymphatic System Could be Driving Force Behind DMD Progression
In two recent studies, a team of scientists under the guidance of Mendell Rimer,...
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Blood Biomarkers Could Offer More Tailored Treatment for Duchenne Muscular Dystrophy
Researchers from the University of Florida and Leiden University Medical Center (LUMC) have discovered...
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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...