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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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Sarepta to Lay Off About 500 Employees After Duchenne Gene Therapy Setbacks
Sarepta stated that company intends to halt development on a number of medicinal products...
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Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform...
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Sarepta Therapeutics Halts ELEVIDYS Shipments for Non-Ambulatory DMD Patients
Due to two documented incidents of severe liver failure that resulted in death, Sarepta...
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JCR Pharmaceuticals Proprietary JUST-AAV Capsids May be Used in the Development of Duchenne Cures
Alexion, AstraZeneca’s rare disease subsidiary, is licensing specialized gene therapy vehicles from the Japanese...
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Taiho Pharmaceutical Announced TAS-205 Phase 3 Clinical Study Did Not Meet Its Primary Endpoint
Taiho Pharmaceutical announced that the results of a phase III, randomized, placebo-controlled, double-blind and...
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Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies
Edgewise Therapeutics, a leading muscle disease biopharmaceutical company, today unveiled positive results in its...
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Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform...
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Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
As part of the FDA’s ongoing review, the Company has been informed that an...
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Cumberland’s Ifetroban Demonstrated a 5.4% Improvement Compared to Placebo-Treated Patients
Cumberland Pharmaceuticals a specialty pharmaceutical company committed to developing new products for rare diseases,...
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Capricor Therapeutics Announces Positive 4-Year Data of Deramiocel (CAP-1002) in Duchenne Muscular Dystrophy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases,...
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Capricor Therapeutics Announces BMD Orphan Drug Designation and DMD Program Regulatory Development for Deramiocel (CAP-1002)
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment...
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Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients
ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy,...
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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...