Duchenne Muscular Dystrophy

Latest News

An analysis of outcomes from part 2 of the EMBARK trial included 14 patients who had received a placebo in part 1 of the study and were aged 8 to 9 years at crossover. | Image Credit: RFBSIP - stock.adobe.com
DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

June 13th 2025

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD).

With approved gene therapies available for patients 4 years and older, the question of how early detection will impact the use of gene therapy in these patients remains. | Image credit: ChiccoDodiFC - stock.adobe.com
Navigating Hope, Risk, and the Unknown in a Changing DMD Treatment Landscape

May 30th 2025

Based on western blot testing, patients who received gene therapy at 2 years old showed a mean dystrophin expression of 93.87% of normal. | Image Credit: Treecha - stock.adobe.com
DMD Gene Therapy Shows Efficacy, Safety in Patients Treated at 2 Years Old

May 23rd 2025

While patients should be closely monitored in the first 90 days post-treatment, delandistrogene moxeparvovec showed a manageable, consistent safety profile in a broad population of patients. | Image Credit: Hypnosis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 16th 2025

Quantitative magnetic resonance imaging seemed to show that delandistrogene moxeparvovec protects muscle from progressive damage in DMD. | Image credit: OlegKachura - stock.adobe.com
Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

May 14th 2025

More News

Sarepta Therapeutics Inc | Image Credit: Timon - stock.adobe.com
January 5th 2024

Sarepta Therapeutics Seeks to Expand Delandistrogene Moxeparvovec Indications

Maggie L. Shaw
Delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) is currently approved to treat Duchenne muscular dystrophy in 4- and 5-year-old patients who have a confirmed DMD gene mutation.
Human lungs on scientific background | Image Credit: Crystal light - stock.adobe.com
January 2nd 2024

Proposed Guidance for DMD-Related Respiratory Function Evaluation in the UK

Maggie L. Shaw
A multidisciplinary team has proposed respiratory care guidance for pediatric and adult patients living with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK), with these recommendations meant to optimize DMD care day to day and in acute episodes.
child in wheelchair and parent | Image credit: Art_Photo - stock.adobe.com
December 29th 2023

Descriptions of Physical Limitations, Symptoms of DMD From the Patient, Caregiver Perspective

Skylar Jeremias
A recent study encapsulates how patients and caregivers characterize the impact of Duchenne muscular dystrophy (DMD) on patients’ physical limitations and symptom burden, potentially helping to inform patient-centered strategies for assessing clinical outcomes in DMD research.
Graphic representing 2023's top 5 DMD content
December 21st 2023

Top 5 Most-Read DMD Articles of 2023

Maggie L. Shaw
This year’s top content in the Duchenne muscular dystrophy (DMD) space focused on a novel gene therapy approval, the high cost of care for this genetic disorder, and steps forward in other treatments.
Medical research, phases of vaccine clinical trials | Image Credit: Olivier Le Moal - stock.adobe.com
December 15th 2023

FDA Extends Review Time for Investigational Duchenne Agent Givinostat

Marco Meglio
Givinostat, a histone deacetylase inhibitor from Italfarmaco, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial investigating the agent vs placebo.
Icon representing data analysis | Image Credit: ipopba - stock.adobe.com
December 7th 2023

New Investigation Analyzes Magnetic Resonance Measures of DMD for Disease Progression

Maggie L. Shaw
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

Marco Meglio
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

Maggie L. Shaw
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

Maggie L. Shaw
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

Maggie L. Shaw
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

Erin Hunter
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

Maggie L. Shaw
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

Maggie L. Shaw
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

Pearl Steinzor
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

Mary Caffrey
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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