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
Belief BioMed Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate BBM-D101
Belief BioMed Reports FDA IND Clearance for Gene Therapy Candidate BBM-D101 for Duchenne Muscular...
Press Releases
FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)
Nippon Shinyaku and the National Center of Neurology and Psychiatry jointly discovered the antisense...
Press Releases
3 Drugs Receiving Accelerated Approval from the FDA and Reviewed by the Office of Inspector General Raised Concerns
Despite shaky evidence and reviewer differences, the US Food and Drug Administration swiftly approved...
Press Releases
Sarepta Reiterated Total Net Product Revenue of $3.0 Billion in 2025
As children and families battling Duchenne Muscular Dystrophy wait for drug prices to drop,...
Press Releases
Wave Life Sciences Announces Plans for Exon 53 Skipping Study in 2025
Alpha-1 antitrypsin deficiency (AATD), Huntington's disease (HD), obesity, and Duchenne muscular dystrophy (DMD) are...
Press Releases
Edgewise Therapeutics Announces Plans for 2025
Edgewise Therapeutics has announced its 2025 plans for Duchenne muscular dystrophy. The company said...
Press Releases
Plans for DYNE-101 in DM1 and DYNE-251 in DMD Are Announced by Dyne Therapeutics
Dyne Therapeutics announced positive clinical data for its therapies DYNE-101 and DYNE-251 during an...
Press Releases
Avidity Biosciences Prepares for Three Rare Muscle Disease Programs’ 2025 BLA Submission and Commercialization
A biopharmaceutical firm dedicated to developing a novel class of RNA treatments known as...
Press Releases
Percheron Disappointed in Avicursen (ATL1102) Study For Duchenne (DMD)
Percheron Therapeutics has announced that it will investigate avicursen (ATL1102) in other indications besides...
Press Releases
Santhera Enters into Supply and Distribution Agreement for AGAMREE (Vamorolone) with Clinigen Group
Santhera Pharmaceuticals announces the signing of an exclusive agreement with Clinigen Group to manage...
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...