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Press Releases
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 with Duchenne muscular dystrophy (DMD). The 24-month open-label extension data focused on Upper Limb Function and showed that patients who started Deramiocel after one year on placebo experienced a significantly slower rate of upper limb...
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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 Duchenne muscular dystrophy (Duchenne) that uses small activating RNA (saRNA) to increase the production of utrophin. The Phase I study data were presented at the 31st Annual Congress of the World Muscle Society (WMS 2026)...
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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...
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Satellos Completes Enrollment in Phase 2 Forazapadin Trial for Duchenne Muscular Dystrophy
Satellos Bioscience has announced the completion of enrollment in BASECAMP, a Phase 2 clinical...
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Exegenesis Bio and Modalis Collaborate to Advance MDL-201 for Duchenne Muscular Dystrophy
Exegenesis Bio and Modalis Therapeutics Corporation have announced a research collaboration and license agreement...
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Satellos’ Duchenne Drug SAT-3247 Receives Official WHO Name: Forazapadin
Satellos Bioscience has announced that its investigational Duchenne muscular dystrophy (DMD) treatment SAT-3247 has...
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FDA Accepts Del-zota Application for Priority Review in DMD44
The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA)...
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NewBiologix and Synastra Partner to Advance DMD Gene Therapy Manufacturing
A new international collaboration is bringing together biotechnology expertise from Switzerland and Turkiye to...
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ANVISA Cancels Elevidys Registration in Brazil
Brazil’s National Health Surveillance Agency (ANVISA) has cancelled the registration of Elevidys, a gene...
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FDA Allows Expanded Enrollment in Signature Biologics’ DMD Cell Therapy Study
Signature Biologics has announced that the U.S. Food and Drug Administration (FDA) has allowed...
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IPS HEART Aims to Restore Full-Length Dystrophin with Two Regenerative Cell Therapies for Duchenne Muscular Dystrophy
IPS HEART has announced another important regulatory milestone after receiving FDA Rare Pediatric Disease...
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GEN6050X Exon 50 Skipping Base Editing Drug: One-Year Results Show Encouraging Progress for Duchenne Muscular Dystrophy
The GEN6050X exon 50 skipping base editing drug, developed by GenAssist Therapeutics, is attracting...
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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...
Press Releases
Satellos Completes Enrollment in Phase 2 Forazapadin Trial for Duchenne Muscular Dystrophy
Satellos Bioscience has announced the completion of enrollment in BASECAMP, a Phase 2 clinical...
Press Releases
STM-01 Enters Phase 2 Clinical Trial for Duchenne Muscular Dystrophy
Secretome Therapeutics has announced the start of THRIVE-2, a Phase 2 clinical study evaluating...
Care Guidelines
Duchenne Muscular Dystrophy Speech Delay: Understanding Language Development in Children with DMD
Duchenne muscular dystrophy speech delay is an important developmental concern that families and healthcare...