Editorials
The Systemic Failure of DMD Associations: Why Duchenne Patients Are Still Waiting
Duchenne muscular dystrophy (DMD) is not a newly discovered condition, nor is it an unsolvable one. Scientific progress over the past two decades has produced exon-skipping therapies, corticosteroid optimization protocols, and most notably, gene therapies that are already approved in major regulatory jurisdictions. Yet, across large parts of the...
Editorials
In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?
Elevidys (delandistrogene moxeparvovec) is a gene therapy developed for Duchenne muscular dystrophy (DMD). It was first approved in the United States and has subsequently received regulatory approvals in several other countries. However, Elevidys is not currently approved in the European Union.In Which Countries Is Elevidys Approved?Is Elevidys Approved in...
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Editorials
Why Isn’t Roche Applying Marketing Authorization in Every Country for Elevidys Gene Therapy?
Gene therapies, particularly those like Elevidys, have the potential to revolutionize the treatment of...
Editorials
Frequently Asked Questions About Solid Biosciences SGT-003 Gene Therapy
Many research companies around the world are researching treatments for Duchenne, one of the...
Editorials
Our Call to Anvisa: All Brazilian Children Over 7 Should Receive Elevidys Gene Therapy
Duchenne Muscular Dystrophy (DMD) is a rare, life-limiting genetic disorder that primarily affects boys,...
Editorials
Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments
While DMD families struggle to restore their children to health, pharmaceutical companies whose primary...
Editorials
The Challenge of Accessing DMD Gene Therapy: Is Geography Destiny?
Why are DMD gene therapies so expensive? Over the last decade, significant advancements have...
Editorials
Call from Families to European Medicines Agency (EMA): Re-examine Ataluren (Translarna)
Three parents representing 800 families and their children affected by Duchenne Muscular Dystrophy and...
Editorials
Brazilian Children Over 7 and Their Families Demand Elevidys Gene Therapy from Anvisa
ELEVIDYS, the only currently approved gene therapy for DMD, is approved in Brazil for...
Editorials
Will Elevidys Receive Approval from the European Medicines Agency (EMA)?
Elevidys, a gene therapy developed by Sarepta Therapeutics for the treatment of Duchenne muscular...
Editorials
Turkish DMD Patients Who are Eligible for Exon 51 Skipping Treatment (Eteplirsen) Request Their Medications from the Ministry of Health
The DMD Dayanisma Community, founded by families of children with Duchenne Muscular Dystrophy and...
Editorials
First Girl in the World to Receive Elevidys Gene Therapy: Raniya Scott
When Raniya Scott was just two years old, her world changed with an unexpected...
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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,...
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...