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
BBM-D101 Gene Therapy Phase 1/2 Clinical Trial Begins
Belief BioMed (BBM), headquartered in China, is a global innovative biotechnology company integrating the...
Research
RGX-202 Gene Therapy Phase 3 Clinical Trial Begins
RGX-202, one of the alternative gene therapy candidates developed for Duchenne Muscular Dystrophy, continues...
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Entrada Therapeutics Begins Clinical Trials in Europe for Exon 44 and Exon 45 Skipping Therapies
Entrada Therapeutics has officially launched clinical trials in Europe for its innovative exon skipping...
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Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model
Preclinical results demonstrating the potential of Tevard Biosciences' tRNA therapy for Duchenne Muscular Dystrophy...
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Satellos Prepares to Launch Phase 2 Clinical Study of SAT-3247 for the Treatment of Duchenne Muscular Dystrophy
Satellos Bioscience, a clinical-stage biotechnology company focused on developing regenerative therapies for muscle diseases,...
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mRNA Studies in DMD Treatment: Future Therapeutic Candidates
Given that FDA-approved exon skipping therapies and gene therapy for Duchenne Muscular Dystrophy, which...
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Miraculous Steroid Vamorolone (Agamree) Could Offer Safer Treatment for DMD
A novel steroid medication that is used to treat Duchenne muscular dystrophy has showed...
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Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers
Keros Therapeutics, a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to...
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Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
Duchenne Exon 51 skipping therapies are innovative treatments aimed at addressing the genetic mutation...
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Shanghai Jiao Tong University School of Medicine Begins Clinical Trials of LE051 Exon 51 Skipping Therapy
The purpose of LE051 study is to evaluate the safety, tolerability, and efficacy of...
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Fn14 Protein May Protect Muscle Stem Cells, Offering Promise for Development of Duchenne Treatments
For millions of people, losing muscle means losing their freedom as well as their...
Research
Significant Improvements Seen in 3 Children Receiving Dystrogen Therapeutics’ DT-DEC01 Cell Therapy
In non-ambulatory individuals with Duchenne muscular dystrophy (DMD), DT-DEC01 (Dystrogen Therapeutics), an experimental dystrophin...
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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...