Editorials
7 Breakthrough DMD Research Programs That Could Transform Duchenne Treatment in 2027
Duchenne muscular dystrophy (DMD) research continues to advance, opening new possibilities for people living with the condition. While gene therapy has attracted significant attention in recent years, it is only one of many approaches being explored. Today's DMD research programs include gene editing, targeted exon skipping, muscle regeneration, cell...
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...
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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...
Editorials
World Duchenne Awareness Day 2026 (#WDAD2026)
Every year, World Duchenne Awareness Day (WDAD) brings the global Duchenne community together to...
Editorials
Do Social Media Hashtag Campaigns Really Help DMD Associations Improve Access to Approved Treatments for DMD Families?
Every year, dozens of Duchenne muscular dystrophy (DMD) associations organize social media campaigns encouraging...
Editorials
If Elevidys Works for Duchenne Muscular Dystrophy, Why Is Sarepta’s Stock Falling? What Families Need to Know Before Choosing Gene Therapy
Few Duchenne muscular dystrophy (DMD) treatments have generated as much excitement, controversy, and debate...
Editorials
Elevidys Under Scrutiny: A Candid Interview with the Turkish Representative of DMDWarrioR on Cost, Effectiveness, and Ethics
Elevidys gene therapy has shown promising results in clinical trials for DMD. However, questions...
Editorials
Vamorolone (Agamree) Rejected by INESSS: When Cost Takes Priority Over Compassion in DMD Care
The recent decision by INESSS (Institut national d'excellence en santé et en services sociaux)...
Editorials
PBGENE-DMD Gene Editing Therapy: Benefits, Eligibility, and FAQs
PBGENE-DMD Gene Editing Therapy is an investigational, first-in-class treatment developed by Precision BioSciences for...
Editorials
Why CK, AST, and ALT Data Matter More Than Videos in Duchenne Gene Therapy
In the evolving landscape of gene therapy for Duchenne muscular dystrophy (DMD), one issue...
Editorials
Duchenne Muscular Dystrophy Treatment Access: A Global Failure of Equity, Policy, and Accountability
Duchenne muscular dystrophy (DMD) is no longer a condition defined solely by its biology....
Editorials
Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
The landscape of potential new gene therapies for Duchenne muscular dystrophy (DMD) is rapidly...
Editorials
The Brutal Truth: Why Duchenne Muscular Dystrophy Treatment Costs Are Out of Control
The duchenne muscular dystrophy treatment cost has become one of the most controversial and...
Editorials
The Accountability Crisis in Duchenne Muscular Dystrophy (DMD) Associations: Why Donations Must Lead to Measurable Outcomes
Where do DMD associations spend the donations they receive, and how much of it...
Latest articles
Editorials
7 Breakthrough DMD Research Programs That Could Transform Duchenne Treatment in 2027
Duchenne muscular dystrophy (DMD) research continues to advance, opening new possibilities for people living...
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...
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...