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...
Press Releases
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
ImmunoForge Receives FDA IND Approval for Phase 2 Clinical Trial of ‘Pemziviptadil’ for DMD Cardiomyopathy
A medication for DMD cardiomyopathy, pemziviptadil is used once a week and is based...
Research
Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne
The National Center of Neurology and Psychiatry is announced the publication of a research...
Research
What is Ataluren (Translarna)? What is Ataluren Used For?
Ataluren (Translarna) is an investigational drug used to delay disease progression in outpatients with...
Research
What is Deramiocel? What Does CAP-1002 Do?
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment...
Research
Is Duchenne Gene Therapy a Suitable Treatment Despite its Immunogenic Class Effect?
Progressive muscle weakness and eventual mortality as a result of cardiomyopathy or respiratory complications...
Research
New Research on Duchenne Muscular Dystrophy Protein Interactions Lead to More Targeted Therapies
A pioneering discovery has shown the complicated relationships between dystrophin, a muscle stability protein,...
Research
Zebrafish Modeling Shows Promise in Duchenne (DMD) Research
Zebrafish (Danio rerio) has emerged as a valuable model organism for investigating musculoskeletal development...
Research
University of Miami Health System and Holtz Children’s Hospital Physicians Evaluated the Effectiveness of ELEVIDYS
University of Miami Health System and Holtz Children’s Hospital physicians are playing a major...
Research
SPLICER Gene Editing Tool Holds A Lot of Potential to Treat Duchenne Muscular Dystrophy
A new gene editing tool that helps cellular machinery skip parts of genes responsible...
Research
Researchers Discover 18-Digit Code (Wnt7a) that Allows Proteins to Hitch a Ride Around the Body
Researchers from the University of Ottawa and The Ottawa Hospital have found an 18-digit...
Research
Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment...
Research
Satellos Produced Near-Normal Levels of Dystrophin in Experiment with Dogs with DMD with SAT-3247
According to the announcement made by Satellos Bioscience Inc., in an experiment conducted on...
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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,...
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...
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...
Press Releases
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...
Press Releases
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...