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
Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease
Cumberland Pharmaceuticals, a specialty pharmaceutical company with development efforts focused on new products for...
Research
A Novel “Mini-CRISPR” Could Be More Successful at Editing the Dystrophin Gene
Several research groups have been attempting to create compact CRISPR systems that can fit...
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Everything You Should Know About Genethon’s Gene Therapy GNT0004 for Duchenne Muscular Dystrophy
The pivotal phase of the trial, which is scheduled to begin in Q2 2025,...
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Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD)
Today, Roche revealed encouraging topline data from the second year of the EMBARK trial,...
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Personalized Miniorgans could Speed up Treatment of Duchenne
Personalized antisense oligonucleotides (ASOs) may accelerate studies on treating Duchenne muscular dystrophy (DMD).Every year,...
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Next Generation Lipid Nanoparticles (LNPs) for the Treatment of Duchenne
Delivering the dystrophin protein to the afflicted muscles is one of the most difficult...
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What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy?
Bobcat mRNA works differently than exon skipping or AAV-microdystrophy mechanisms. This next-generation method could...
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Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area...
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Insmed’s Gene Therapy (INS1201) Poised to Challenge Duchenne Muscular Dystrophy (DMD)
Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne...
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Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy
Solid Biosciences shared that four patients have been dosed in the SGT-003 gene therapy...
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Dr. Liubov Gushchina’s Exon 17 Skipping Study Rewarded with Grant
We are happy to present the winners of the American Neuromuscular Foundation's (ANF) 2024...
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The FDA Designates IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy an Orphan Drug
The FDA has granted IPS HEART Orphan Drug Designation (ODD) for GIVI-MPCs because of...
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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...
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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...
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...