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
NSAA Alone Is Not Enough: Why Creatine Kinase (CK), AST, ALT, and Dystrophin Levels Should Be Disclosed
So why do many DMD clinical trials avoid clearly reporting these key biomarkers? This...
Editorials
Genethon Confirms Two-Year Efficacy of GNT0004 Gene Therapy with Improvements in Creatine Kinase (CK) Levels and NSAA Scores
Gene therapy research for Duchenne muscular dystrophy (DMD) continues to advance. At the MDA...
Editorials
Compared to Its Million-Dollar Price Tag, How Much Does Elevidys Gene Therapy Improve the Condition of Children With DMD?
Elevidys, the only gene therapy approved by the FDA for Duchenne muscular dystrophy (DMD),...
Editorials
SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin
SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by...
Editorials
‘One-Time’ Gene Therapies or Exon-Skipping Therapies: Which May Be Better for Duchenne’s Disease Today?
Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), aprogressive muscle...
Editorials
Creatine Kinase (CK), Dystrophin Level and NSAA Score in Elevidys Gene Therapy
It remains a matter of curiosity how much Creatine Kinase (CK) levels decrease, NSAA...
Editorials
Why Does the FDA Approve DMD Therapies That Fail in Clinical Trials?
Do gene therapies, marketed for millions of dollars, and exon-skipping therapies administered monthly and...
Editorials
Why Elevidys Was Not Approved by the European Medicines Agency (EMA)?
The Elevidys (active substance: delandistrogene moxeparvovec) gene therapy was not approved by the European Medicines...
Editorials
Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production?
Duchenne Muscular Dystrophy (DMD) is a severe, inherited neuromuscular disorder that leads to progressive...
Editorials
Could Axolotl’s Self Regenerative Ability Be Effective in Treating Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is a severe X‑linked genetic disorder characterized by absence or...
Editorials
Del-Zota DMD RNA Therapy from Avidity Biosciences Reverses the Progression of Disease
Due to the deaths of two patients following the use of Elevidys, a DMD...
Editorials
Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States
According to the Swiss pharmaceutical company, the halt, which goes into effect on 22nd...
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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...