HomePress Releases
Press Releases
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, 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...
Keep exploring
Press Releases
Avidity Biosciences Announces U.S. Managed Access Program (MAP) for Investigational Therapy del-zota (Exon 44 Skipping)
Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota)...
Press Releases
Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature
Satellos announced the publication in Nature Communications of new research from a scientific team...
Press Releases
Solid Biosciences Awarded Innovation Passport Designation Under the UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Solid Biosciences announced that SGT-003, the Company’s investigational gene therapy for Duchenne muscular dystrophy...
Press Releases
Sarepta’s DMD Exon-Skipping Therapies Fail Confirmatory Study, Stock Price Down
Sarepta Therapeutics' difficulties only get worse. In patients with Duchenne muscular dystrophy, the business...
Press Releases
PPMD Provides $400,000 in Funding to MyoGene Bio for MyoDys Gene Editing Platform
PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization's...
Press Releases
Solid Biosciences Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions
Solid Biosciences announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical...
Press Releases
Five-Year Data of AGAMREE (vamorolone) in Patients with DMD Show Improved Safety Profile with Comparable Effectiveness to Standard of Care Corticosteroids
Santhera reported positive topline results from an analysis of long-term data, including first assessments...
Press Releases
Capricor Therapeutics Publishes Peer-Reviewed Study About Deramiocel Cell Therapy
Capricor Therapeutics announced the publication in Biomedicines, an international peer-reviewed journal, describing a novel...
Press Releases
REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program
REGENXBIO announced the completion of enrollment in the AFFINITY DUCHENNE pivotal trial of RGX-202,...
Press Releases
Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy
SRP-9001-104 in Duchenne muscular dystrophy (DMD): Topline results were reported for three patients with...
Press Releases
Avidity Biosciences to be Acquired by Novartis
Avidity Biosciences, which is developing an exon 44 skipping therapy for Duchenne muscular dystrophy,...
Press Releases
A Statement from NorthStar Network on Givinostat (Duvyzat)
According to an announcement on the Muscular Dystrophy UK website, NorthStar Network stated: 'As...
Latest articles
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...
Press Releases
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...
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...