Duchenne Muscular Dystrophy

Latest News
June 13th 2025

DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

May 30th 2025

Navigating Hope, Risk, and the Unknown in a Changing DMD Treatment Landscape

May 23rd 2025

DMD Gene Therapy Shows Efficacy, Safety in Patients Treated at 2 Years Old

May 16th 2025

Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 14th 2025

Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

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April 2nd 2025

The Value of Newborn Screening for DMD: Barry Byrne, MD, PhD

May 26th 2024

Dr Migvis Monduy on the Most Promising Areas of DMD Research

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July 18th 2024

Insurance Insights: Dr Jason Shafrin Estimates DMD Insurance Value

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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
April 18th 2025

Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery

Rose McNulty
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
April 18th 2025

At-Home DMD Video Assessment Found Valid, Reliable

Rose McNulty
The remote measurement tool enables Duchenne muscular dystrophy (DMD) assessments through analysis of caregiver-recorded videos.
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
April 11th 2025

Eteplirsen Treatment Linked to Slower Cardiac Decline in DMD

Rose McNulty
None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.
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
March 27th 2025

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

Rose McNulty
WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial.
Real-world data suggest delandistrogene moxeparvovec is well-tolerated and effective in patients with DMD. | Image credit: natali_mis - stock.adobe.com
March 26th 2025

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

Rose McNulty
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.
Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy approved for the treatment of DMD. Image credit: natali_mis - stock.adobe.com
March 21st 2025

Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD

Rose McNulty
Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows Duchenne muscular dystrophy (DMD) progression.
Multiparametric qMRI could potentially help differentiate between DMD and BMD early and improve the management of these conditions. | Image credit: megaflopp - stock.adobe.com
March 18th 2025

MRI Could Aid in Early Differential Diagnosis of DMD, BMD

Rose McNulty
Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.
Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy or Becker muscular dystrophy. | Image credit: Nana_studio - stock.adobe.com
February 28th 2025

Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood

Rose McNulty
Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life.
In the INSPIRE DUCHENNE trial, interim 90-day biopsy data showed an average microdystrophin expression of 110% among the first 3 participants in the study. | Image credit: RFBSIP - stock.adobe.com
February 26th 2025

SGT-003 Gene Therapy Shows Potential in DMD

Rose McNulty
In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants with Duchenne muscular dystrophy (DMD) at 90 days post treatment.
Vertebral fractures are common in glucocorticoid-associated osteoporosis and are a clinical indicator of osteoporosis in young patients. | Image credit: Hugnaka - stock.adobe.com
February 14th 2025

Adults With DMD More Than Twice as Likely to Experience Fractures

Rose McNulty
Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of osteoporosis in adult men with Duchenne muscular dystrophy (DMD).
Ifetroban may improve LVEF in patients with DMD. - REDPIXEL - stock.adobe.com
February 7th 2025

Ifetroban Improves Heart Function in DMD

Rose McNulty
There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results suggest ifetroban may improve left ventricular ejection fraction in patients with DMD.
Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com
January 28th 2025

EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD

Rose McNulty
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).
Heart and stethoscope | Image credit: inthasone - stock.adobe.com
January 24th 2025

Prophylactic Cardiac Medication May Prolong Survival in DMD

Rose McNulty
Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found.
AJMC
January 14th 2025

Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy

Jason Shafrin, PhD Suhail Thahir, MS Alexa C. Klimchak, MA Ivana Audhya, MSc Lauren E. Sedita, MS John A. Romley, PhD
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 per year.
Past studies have explored the association between motor and cardiac function in younger patients with DMD, but there is a lack of research on this relationship as patients with DMD age. | Image credit: Nana_studio - stock.adobe.com
January 13th 2025

Later Loss of Ambulation Linked With Better Heart Health in DMD

Rose McNulty
The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).
lab test tubes | Image credit: kwanchaift – stock.adobe.com
December 22nd 2024

Study Highlights Trends, Emerging Innovations in DMD Research

Hayden E. Klein
An analysis revealed global trends and emerging focus areas in Duchenne muscular dystrophy (DMD) research, emphasizing the growing impact of precision medicine and gene therapies.
Heart anatomy on paper | Image credit: larisikstefania – stock.adobe.com
December 20th 2024

MRI Biomarker May Signal Early Heart Damage in DMD

Hayden E. Klein
Septal circumferential strain (Ecc) was significantly decreased in LGE-negative boys with Duchenne muscular dystrophy (DMD) and correlated to ventricle changes.
Gene therapy | Image credit: Treecha – stock.adobe.com
December 19th 2024

FDA Pauses PepGen’s DMD Drug Trial in the US

Hayden E. Klein
The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.
The top 5 DMD content of 2024
December 19th 2024

Top 5 Duchenne Muscular Dystrophy Content of 2024

Hayden E. Klein
From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and research.
Gene therapy graphic with DNA strands | Image credit: natali_mis – stock.adobe.com
November 25th 2024

Real-World Challenges in DMD PMO Treatment

Hayden E. Klein
A claims-based study revealed high adherence to phosphorodiamidate morpholino oligomer (PMO) treatments for Duchenne muscular dystrophy (DMD), but highlights limitations in interpreting real-world effectiveness from claims data.
Boy with DMD using walker | Image credit: Svitlana – stock.adobe.com
November 23rd 2024

Viltolarsen Slows DMD Progression in Small Study

Hayden E. Klein
Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).
FDA website | Image Credit: © Postmodern Studio - stock.adobe.com
November 22nd 2024

FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy

Marco Meglio
The new drug application (NDA) includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.
Researcher working with microplate in the laboratory | Image credit: angellodeco – stock.adobe.com
November 14th 2024

Sarepta Halts Development, Testing of DMD Therapy SRP-5051

Hayden E. Klein
Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.
Gene therapy research | Image credit: RFBSIP – stock.adobe.com
Published: October 29th 2024 | Updated: October 30th 2024

A Year of DMD Gene Therapy Trial Failures

Hayden E. Klein
The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.
Tired caregiver | Image credit: butsaya33 – stock.adobe.com
October 28th 2024

Studies Highlight Heavy Burden on Caregivers of Patients With DMD

Hayden E. Klein
Caregivers of both adults and children with Duchenne muscular dystrophy (DMD) face significant disruptions to their work productivity and personal lives, underscoring the need for better treatments and support systems.
Zinc supplement | Image credit: Celt Studio – stock.adobe.com
October 25th 2024

Zinc Supplements Fall Short for Boys With Duchenne Muscular Dystrophy

Hayden E. Klein
Standard zinc supplementation had limited impact on muscle health or zinc levels in boys with Duchenne muscular dystrophy, highlighting the need for more tailored nutritional strategies.
Nurse helping patient use walker | Image credit: LIGHTFIELD STUDIOS – stock.adobe.com
October 9th 2024

Genotypes May Influence When Patients With DMD Have Loss of Ambulation

Hayden E. Klein
A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).
Currently, 4 exon-skipping drugs are FDA approved for DMD: eteplirsen (Exondys 51), golodirsen (Vyondys 53), casimersen (Amondys 45), and viltolarsen (Viltepso) | Image credit: CrazyJuke - stock.adobe.com
September 26th 2024

WVE-N531 Shows Promising Efficacy in DMD at Interim FORWARD-53 Analysis

Rose McNulty
In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
The authors of the case series noted that prevalent comorbidities among patients with DMD increase the risk of arrhythmias even further. | Image credit: Elena - stock.adobe.com
September 18th 2024

Atrial Arrhythmias Common in DMD, but More Data on Treatment Are Needed

Rose McNulty
Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and more long-term data on the management of arrhythmias in DMD are needed.
When there are changes in muscle microstructures, acoustic attenuation occurs, which may hold potential as an imaging biomarker for DMD. | Image credit: hafakot - stock.adobe.com
September 6th 2024

Novel Imaging Algorithm Shows Promise in DMD Evaluation

Rose McNulty
Measurements on a standard phantom and a clinical data set of patients with Duchenne muscular dystrophy (DMD) were used to validate a novel robust reference frequency method approach, which outperformed typical imaging strategies.
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