Duchenne Muscular Dystrophy

Latest News

The consensus guidelines aim to provide practical guidance for institutions administering gene therapy in a commercial or trial setting. | Image credit: CrazyJuke - stock.adobe.com

Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery

By Rose McNulty

April 18th 2025

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

While previous research on the DVA focused on task-specific measurements, the new study focused on total composite severity percentage among patients with DMD. | Image credit: ChiccoDodiFC - stock.adobe.com

At-Home DMD Video Assessment Found Valid, Reliable

By Rose McNulty

April 18th 2025

None of the eteplirsen-treated patients reached an LVEF below 50%, compared with 22.1% of patients in the control group. | Image credit: inthasone - stock.adobe.com

Eteplirsen Treatment Linked to Slower Cardiac Decline in DMD

By Rose McNulty

April 11th 2025

All 11 participants in the trial have advanced to the extension portion of the study and are receiving WVE-N531 monthly. | Image credit: OlegKachura - stock.adobe.com

WVE-N531 Shows Functional Benefits, Muscle Damage Reversal in DMD

By Rose McNulty

March 27th 2025

Real-world data suggest delandistrogene moxeparvovec is well-tolerated and effective in patients with DMD. | Image credit: natali_mis - stock.adobe.com

Real-World Study Suggests DMD Gene Therapy Is Safe, Effective

By Rose McNulty

March 26th 2025

More News

Icon representing data analysis | Image Credit: ipopba - stock.adobe.com

December 7th 2023

New Investigation Analyzes Magnetic Resonance Measures of DMD for Disease Progression

Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of magnetic resonance measures of muscle quality at different disease stages.

Duchenne medicial concept | Image Credit: hafakot - stock.adobe.com

November 22nd 2023

EMBARK Data Support Delandistrogene Moxeparvovec for DMD Across All Study End Points

Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.

DNA molecules | Image Credit: vitstudio - stock.adobe.com

November 17th 2023

Data on Delandistrogene Moxeparvovec Can Inform Future Gene Therapies

The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.

Duchenne MD green ribbon

November 15th 2023

Heart Failure Undertreated in Duchenne Muscular Dystrophy

With improved survival becoming more common among individuals who have Duchenne muscular dystrophy (DMD), cardiomyopathy is also increasing in prevalence among this population.

DNA concept art | Image credit: nobeastsofierce - stock.adobe.com

November 9th 2023

Key Insights Into BMD, DMD Could Revolutionize Clinical Care and Trials

This retrospective review compared appendicular lean mass (ALM) and ALM index among patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

The disease is characterized by the regression of muscle function, and patients with the rare, fatal disorder will slowly lose ambulation and suffer from respiratory failure | Image Credit: Dr_Microbe - stock.adobe.com

November 2nd 2023

FDA Approves Vamorolone to Treat Duchenne Muscular Dystrophy

The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.

Health Cost Representation Concept | Image Credit: A stockphoto - stock.adobe.com

October 25th 2023

Exon-Skipping Therapy for DMD Linked to High Care Costs, Health Care Resource Utilization

This comprehensive comparative analysis examined the economic and health care resource utilization implications of initiating glucocorticoid and exon-skipping therapy for Duchenne muscular dystrophy (DMD).

Aravindhan Veerapandiyan, MD | Image Credit: Arkansas Children's Hospital

October 21st 2023

Phase 1/2 Findings Highlight RGX-202’s Safety, Impact on Key Duchenne Biomarkers

Isabella Ciccone, MPH

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.

Duchenne medicial concept | Image Credit: hafakot - stock.adobe.com

October 19th 2023

Case Report of Patient Death Following High-Dose Gene Therapy for DMD

In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.

Sarah Boyce, president and CEO, Avidity Biosciences | Image Credit: Avidity Biosciences

October 9th 2023

Avidity Biosciences Gains FDA Orphan Drug Designation for Patients With DMD

Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).

FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy

June 22nd 2023

FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.

image of children

September 21st 2022

Balancing Hopes for a Cure for SMA, DMD With Realistic Expectations

Jared Kaltwasser

A new study looks at the balancing act physicians and families must carry out when managing expectations at a time of great scientific advancement in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs

September 12th 2020

Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs

Jaime Rosenberg

Researchers provided an overview of what they deem a hopeful future for spinal muscular atrophy and Duchenne muscular dystrophy.

Researchers Try to Update Prevalence of Duchenne Muscular Dystrophy

June 15th 2020

Researchers Try to Update Prevalence of Duchenne Muscular Dystrophy

Skylar Jeremias

Studies with better data are still needed to fully understand the epidemiology of Duchenne muscular dystrophy (DMD), according to a recently published review.

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