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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, 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
Santhera Secures Agreement with GEN for the Distribution of AGAMREE (Vamorolone) in Turkiye
Santhera Pharmaceuticals announces the signing of an exclusive agreement with Gen İlaç ve Sağlık...
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Keros Therapeutics Focuses on Advancing KER-065 for Duchenne Muscular Dystrophy (DMD)
Keros Therapeutics announced a strategic realignment designed to reallocate resources towards the development of...
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Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD)
Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough...
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High Anti-AAV Antibodies May No Longer Be a Barrier to Elevidys Administration
One of the most significant obstacles to the application of Elevidys gene therapy developed...
Press Releases
MetrioPharm’s MP1032 Receives EMA Orphan Drug Designation in Duchenne Muscular Dystrophy
MetrioPharm announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD)...
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FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’
Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD) from Sarepta Therapeutics, is now...
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FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys
The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy...
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The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys
Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of...
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Brazil Suspends Use of ELEVIDYS (delandistrogene moxeparvovec)
The Brazilian Health Regulatory Agency (Anvisa) announced that it has temporarily suspended the marketing,...
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FDA Designates Avidity Biosciences’ Delpacibart Zotadirsen (del-zota) as a Breakthrough Therapy for the Treatment of DMD in Individuals with Mutations That Allow for Exon...
Avidity Biosciences today announced that the U.S. Food and Drug Administration (FDA) has granted...
Press Releases
The FDA Designates PBGENE-DMD as an Orphan Drug by Precision BioSciences to Treat Duchenne Muscular Dystrophy
Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform...
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Sarepta Therapeutics Announces Pause of ELEVIDYS Shipments in the USA
Today, Sarepta Therapeutics notified the U.S. Food and Drug Administration (FDA) of its decision...
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DMD
What Is Duchenne Muscular Dystrophy (DMD)?: 108 FAQs
Duchenne muscular dystrophy (DMD), commonly known as Duchenne, is a rare genetic disorder that...
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...
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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...
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...