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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, evaluating ENTR-601-45 in people with Duchenne muscular dystrophy (Duchenne). The first group of participants showed a favorable safety profile and a small increase in dystrophin production after receiving the initial dose. The findings are an...
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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 Skipping Study for Duchenne muscular dystrophy (DMD). The independent Data Monitoring Committee (DMC) has recommended starting Cohort 3 at 18 mg/kg in the ELEVATE-44-201 clinical trial, following its review of available safety and pharmacokinetic data...
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Press Releases
Solid Biosciences Announces 2026 Outlook for Clinical Trials of Duchenne Gene Therapy SGT-003
In a press release issued by Solid Biosciences, it was stated that "With multiple...
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Edgewise Therapeutics Shared its Priorities for Becker and Duchenne Muscular Dystrophy in 2026
Edgewise Therapeutics provided updates on the Company's clinical programs (Becker Muscular Dystrophy / Duchenne...
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Precision BioSciences Anticipates Beginning Patient Dosing for Duchenne Muscular Dystrophy in The Early Second Quarter of 2026
For the DMD program PBGENE-DMD, Precision BioSciences anticipates starting patient dosing in late first...
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Entrada Therapeutics Announced It will Report Data from its Exon Skipping Studies in 2026
According to Entrada Therapeutics' announcement, "2026 will be a data-rich year for Duchenne muscular...
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Nxera Pharma Licenses Japan and Select Asia-Pacific Rights to Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals
Nxera Pharma (“Nxera” or “the Company; TSE 4565) announces that it has entered an...
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Italfarmaco and JCR Pharmaceuticals Announce Commercialisation Agreement for Duvyzat (Givinostat) in Japan and Strategic Collaboration in Rare Disease
Italfarmaco and JCR Pharmaceuticals announced an exclusive licensing agreement for the development and commercialisation...
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Secretary Kennedy Expands Newborn Screening to Include Duchenne Muscular Dystrophy in USA
Duchenne muscular dystrophy (Duchenne) has been officially added to the Recommended Uniform Screening Panel...
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Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
Atossa Therapeutics announced that the U.S. Food and Drug Administration ("FDA") has granted Rare...
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Satellos Receives FDA and International Clearance to Commence Pediatric Phase 2 Testing of SAT-3247 for Duchenne Muscular Dystrophy
Satellos reported that it has obtained Investigational New Drug (IND) clearance from the U.S....
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Dyne Therapeutics to Apply for Accelerated Approval in Q2 2026 for Exon 51 Skipping Therapy (DYNE-251)
Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its...
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Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy
HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys...
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ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study
Sarepta Therapeutics confirmed that the FDA has sanctioned the start of dosing in Cohort...
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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...
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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...