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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...
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 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
New Hope for Duchenne: Ryoncil Clinical Trial Begins
A new breakthrough may change the future of Duchenne muscular dystrophy. Ryoncil (remestemcel-L-rknd), a...
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Regenxbio Reports New Positive Interim Data From Phase 1/2 Affinity Duchenne Trial Of Rgx-202
REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of...
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Tenaya’s TN-301 Beats Givinostat in Improving DMD Muscle Function
Tenaya Therapeutics presented encouraging preclinical data evaluating TN-301, the company’s highly selective HDAC6 inhibitor,...
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Vamorolone (Agamree) in DMD: Real-World Experience
Santhera Pharmaceuticals announced that full results from long-term real-world comparative analyses, including baseline data...
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Santhera Announces Proposed Inclusion and Reimbursement of AGAMREE (Vamorolone) within Spain’s National Health System for the Treatment of Duchenne Muscular Dystrophy
Santhera Pharmaceuticals announces that the Spanish Interministerial Commission on Prices of Medicines, has proposed...
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Data Monitoring Committee Recommended Initiating an Increased Dose for Group 2 in Entrada’ Exon 44 Skipping Study
Entrada Therapeutics announced that an independent Data Monitoring Committee (DMC), per study protocol, has...
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Satellos Announces First Participant Dosed in Phase 2 Pediatric Study of SAT-3247 for Duchenne Muscular Dystrophy
Satellos Bioscience announced that the first participant has been dosed in BASECAMP, a three-month,...
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Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dystrophy
Precision BioSciences announced that the Company received a Study May Proceed notification from the...
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Solid Biosciences Aligns With FDA on SGT-003 Phase 3 Trial Design for Duchenne Muscular Dystrophy Gene Therapy
Solid Biosciences announced a positive regulatory update from its recent Type C meeting with...
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Sarepta Reported 3-Year Topline Data of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy
Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK...
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Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy
Atossa Therapeutics, announced that the U.S. Food and Drug Administration ("FDA") Office of Orphan...
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AGAMREE (Vamorolone) has been approved in Switzerland by Swissmedic
Santhera Pharmaceuticals announces that the Swiss Agency for Therapeutic Products (Swissmedic), has approved AGAMREE...
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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...