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
Agamree (vamorolone) will be Covered in Slovenia by the Slovenian Health Insurance Fund
The Slovenian Health Insurance Institute (ZZZS) today added Agamree (vamorolone) to the list of...
Press Releases
Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy
According to the Foundation's press office, "as a result of the negotiations, the price...
Press Releases
Belief BioMed BBM-D101 Gene Therapy Receives IND Clearance by National Medical Products Administration (NMPA)
Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today...
Press Releases
New Drug Submission for AGAMREE (vamorolone) Accepted for Priority Review by Health Canada for the Treatment of Duchenne Muscular Dystrophy
Catalyst Pharmaceuticals has announced that Health Canada has accepted their New Drug Submission (NDS)...
Press Releases
Genethon CEO Frederic Revah Announces GNT0004 Gene Therapy Will Provide Amazing Results for DMD (Duchenne Muscular Dystrophy)
The Chief Executive Officer of Genethon, Frederic Revah, made the announcement on the YouTube...
Press Releases
U.S. Department of Defense Announces Duchenne Muscular Dystrophy Research Programs and Funding Opportunities for Fiscal Year 2025
We extend our deepest gratitude to the Members of Congress who fought to preserve...
Press Releases
Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe
Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta...
Press Releases
Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed
In May 2024, PTC Therapeutics had been announced that the European Commission (EC) has...
Press Releases
Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial in DMD Including Significant Functional Benefit and Reversal of Muscle Damage after 48 Weeks...
Wave Life Sciences, a clinical-stage biotechnology company focused on unlocking the broad potential of...
Press Releases
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201
Entrada Therapeutics, announced it has received authorization from the United Kingdom’s Medicines and Healthcare...
Press Releases
Regenxbio Reports Positive Biomarker Data From Affinity Duchenne Trial Of Rgx-202 Gene Therapy
REGENXBIO reported new, positive interim data from two additional patients in the Phase I/II...
Press Releases
Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Satellos Bioscience, a biotech company developing new small molecule therapeutic approaches to improve the...
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...