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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
Precision BioSciences Presents Preclinical Efficacy and Durability Data on PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) at the 2025 Muscular Dystrophy Association...
This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances...
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Sarepta, Reports 16 Years Old Boy Death After Elevidys Gene Therapy Treatment
On Tuesday, Sarepta Therapeutics said that the first recorded fatality associated with their gene...
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Givinostat’s Exclusive Distribution Agreement in 17 Central and Eastern European Countries, Including the Baltic States, has been Announced by Italfarmaco and Medis
Italfarmaco and Medis, a leading pharmaceutical commercialisation company in Central and Eastern Europe, today...
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Biophytis’ BIO101 Therapy for Treatment of Sarcopenia Could Bring Hope to Duchenne Muscular Dystrophy Patients
The Phase 2 clinical trial SARA-INT was published in the Journal of Cachexia, Sarcopenia...
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Eteplirsen (Exondys 51) Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy
Following promising results in slowing heart function decline in patients with DMD, Eteplirsen (Exondys...
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Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy
Positive long-term results from the company's ongoing HOPE-2 open label extension ("OLE") clinical trial...
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ITF Therapeutics LLC Presents Long-Term Data of DUVYZAT™ (givinostat) for Treatment of Duchenne Muscular Dystrophy at MDA Clinical and Scientific Conference
Today, Italfarmaco's U.S. affiliate, ITF Therapeutics LLC, announced seven abstracts for the Muscular Dystrophy...
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Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin
Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin,...
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Roche to Share Ongoing Work on Elevidys Gene Therapy at Muscular Dystrophy Association (MDA) 2025 Conference
At the Muscular Dystrophy Association (MDA) 2025 Conference, Roche will discuss their ongoing work...
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Dyne Therapeutics Announces Unprecedented and Sustained Functional Improvement Through 18 Months from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics, a clinical-stage company focused on advancing life-transforming therapeutics for people living with...
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SonoThera will Present New Data of Genetic Medicine Using Targeted, Ultrasound-Mediated Delivery (UMD) in Treating Duchenne Muscular Dystrophy
SonoThera will present new data at the 2025 MDA Conference demonstrating the potential of...
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Solid Biosciences Plans to Request a Meeting with the FDA in mid-2025 to Discuss Potential Accelerated Approval Pathways for SGT-003
Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular...
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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...
Press Releases
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...