Duchenne Muscular Dystrophy

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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

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

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AJMC

July 10th 2024

Quantifying the Insurance Value for Rare Diseases: Duchenne Muscular Dystrophy

Jason Shafrin, PhD Suhail Thahir, MS Alexa C. Klimchak, MA Ivana Audhya, MSc Lauren E. Sedita, MS John A. Romley, PhD

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear.

DMD, an inherited disorder characterized by progressive muscle weakness, typically manifests in childhood and currently has no cure. | image credit: OlegKachura - stock.adobe.com

July 5th 2024

Appendicular Lean Mass Index, Fat Mass Index Associated With Motor Function in DMD

Both appendicular lean mass and fat mass index accounted for unique variance in motor function after controlling for age in patients with Duchenne muscular dystrophy (DMD).

Gene editing shows promise in Duchenne muscular dystrophy. | Image credit: Degimages - stock.adobe.com

June 29th 2024

Stem Cell–Based Gene Editing Strategy Shows Promise in DMD

Jared Kaltwasser

Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein.

Blue stamp of FDA approval | Image Credit: argus-stock.adobe.com

June 20th 2024

FDA Grants 2 Approvals to Delandistrogene Moxeparvovec for DMD

Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.

Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com

June 19th 2024

Phase 3 CIFFREO Trial of DMD Gene Therapy Misses Primary End Point

Pfizer’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, did not meet its primary end point of improvement in motor function in ambulatory patients with Duchenne muscular dystrophy (DMD).

Dr Migvis Monduy

June 12th 2024

Barriers to DMD Care Access and How Policy Change Can Support Patients: Dr Migvis Monduy

Rose McNulty Hayden E. Klein

Migvis Monduy, MD, medical director of Neuromuscular and Movement Disorders Programs at Nicklaus Children's Hospital, discussed challenges in Duchenne muscular dystrophy (DMD) treatment access and how policy changes may support patients with DMD.

Doctors planning dmd treatment | Image credit: OlegKachura - stock.adobe.com

June 6th 2024

Delaying Loss of Ambulation May Mitigate Worsening Disease Burden in DMD

Slowing the loss of ambulation in patients with Duchenne muscular dystrophy (DMD) may also mitigate worsening disease burden and overall function, according to a pair of posters presented at ISPOR 2024.

Doctor checking person's leg for muscle weakness | Image credit: Danai - stock.adobe.com

May 23rd 2024

Glucocorticoids Reduce Comorbidity Frequency in Adults With DMD, Study Finds

A retrospective analysis found glucocorticoid treatment to reduce comorbidities in adults with Duchenne muscular dystrophy (DMD) and assessed the relationship between anthropometric measures and respiratory function and functional abilities.

Health care costs | Image credit: utah51 - stock.adobe.com

May 17th 2024

Study Highlights Significant Increases in Utilization, Spending on DMD Drugs in Medicaid

The findings add to recent research on the growing utilization, expenditure, and prices of Duchenne muscular dystrophy (DMD) therapies in the current landscape, an area health care policy could potentially address.

Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com

May 10th 2024

Posters Characterize DMD Caregiver Experiences, Impact of Gene Therapy on Caregiving Demands

Posters presented at the ISPOR—The Professional Society for Health Economics and Outcomes Research meeting explored Duchenne muscular dystrophy (DMD) caregiver experiences and gene therapy’s impact on work opportunities for caregivers.

Using virtual reality device in wheelchair | Image credit: petrushin1984 - stock.adobe.com

April 29th 2024

Virtual Reality and Biofeedback Training Appear Effective for DMD, BMD Rehabilitation

A recent study suggests virtual reality and biofeedback training may be feasible and effective for patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

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

April 25th 2024

Patients With DMD Receiving Eteplirsen Show Prolonged Survival, Study Finds

The study provides evidence of survival benefits among patients with Duchenne muscular dystrophy (DMD) receiving eteplirsen compared with the natural history of the condition.

Person in wheelchair | Image credit: Nana_studio - stock.adobe.com

April 19th 2024

Standard Criteria for Loss of Ambulation Needed in DMD

A recent study suggests the differences between ambulation definitions for patients with Duchenne muscular dystrophy (DMD) can impact the identification of ambulant vs nonambulant individuals, and standard criteria across settings are needed.

Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com

April 12th 2024

Glucocorticoids Preserve Late-Stage Functional Abilities in DMD After Loss of Ambulation, Study Finds

A recent study suggests treatment with glucocorticoids after loss of ambulation can preserve late-stage functional abilities, respiratory function, and cardiac function in patients with Duchenne muscular dystrophy (DMD).

Food and drugs administration quality control | Image Credit: wladimir1804 - stock.adobe.com

March 21st 2024

Givinostat Receives Full FDA Approval for Duchenne Muscular Dystrophy

The approval follows fast track, orphan drug, and rare pediatric designations and an original Prescription Drug User Fee Act date of December 21, which the FDA pushed back to March 21 to have more time to review Italfarmaco’s application.

Benjamin N. Rome, MD, MPH | Image Credit: Brigham and Women's Hospital

March 15th 2024

Dr Ben Rome Scrutinizes the FDA’s Accelerated Approval Pathway for DMD Treatments, Calls for Policy Reform

In a recent issue of JAMA, a team led by Benjamin N. Rome, MD, MPH, examined how the FDA’s accelerated approval process has moved 5 genetically targeted treatments for Duchenne muscular dystrophy (DMD) through its pipeline despite limited evidence on their efficacy.

DNA molecule and dollar sign | Image credit: Sergey Nivens - stock.adobe.com

March 14th 2024

Experts Issue Call for Reform to DMD Drug Pricing, Trial Approval Process

Since 2016, 5 targeted treatments have received accelerated approval from the FDA for use in Duchenne muscular dystrophy (DMD), and the total spend for just 3 of them is $3.1 billion.

Duchenne on a computer screen | Image credit: MQ-Illustrations - stock.adobe.com

March 6th 2024

Case Report Warns of Pneumothorax Risk in Duchenne Muscular Dystrophy

The authors of this report stress the importance of awareness of COVID-19–related complications in young patients who have Duchenne muscular dystrophy, paying particular attention to a potential higher risk of respiratory infections in these patients.

Duchenne | Image credit: hafakot-stock.adobe.com

February 28th 2024

Smartphone Tech Reveals Gait Differences in Duchenne Muscular Dystrophy

Outside of a gait laboratory, this investigation compared gait pattern differences between children who have Duchenne muscular dystrophy (DMD) and their typically developing counterparts to gain more real-world data of DMD-associated gait characteristics.

Muscular dystrophy in large letters | Image Credit: Lemau Studio - stock.adobe.com

February 22nd 2024

Strength Training Safely Boosts Muscular Dystrophy Outcomes, but Further Study Needed

Data are scarce regarding the benefits of strength training for muscular dystrophies, for which there are no cures.

Muscular dystrophy paper and stethoscope | Image credit: taqshatuvango - stock.adobe.com.jpeg

February 13th 2024

Positive Phase 2 Data for SRP-5051 in Duchenne Muscular Dystrophy

The Momentum trial from Sarepta Therapeutics is investigating SRP-5051 (vesleteplirsen) for use among male patients aged 8 to 21 years who have Duchenne muscular dystrophy amenable to exon 51 skipping.

Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com

February 5th 2024

Pre-, Postinfusion Protocol Recommendations for Delandistrogene Moxeparvovec Use in DMD

The gene therapy delandistrogene moxeparvovec-rokl, also known as Elevidys (Sarepta Therapeutics) is indicated to treat Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years, and Sarepta recently filed supplemental data with the FDA seeking to expand the labeled indication.

Doctor in lab coat holds stethoscope. The word Duchenne is written next to it | Image Credit: MQ-Illustrations - stock.adobe.com

January 31st 2024

Translating DMD Clinical Trial Findings to Real-World Application Discouraging

This analysis compared outcomes among patients who have Duchenne muscular dystrophy (DMD) and commercial insurance or Medicaid, focusing on how these differ between patients in clinical trials and those who receive treatment post approval.

Doctors planning DMD treatment | Image Credit: OlegKachura - stock.adobe.com

January 24th 2024

DMD Guidance Could Benefit From Update, Experts Say

MEDLINE, the Turning Research Into Practice database, Google Scholar, and organization guidelines were searched for their ongoing applicability to the management of Duchenne muscular dystrophy (DMD) and to determine if there is a need to develop new treatment recommendations.

Hands of a disabled person with muscular dystrophy holding a ventilator | Image Credit: ins - stock.adobe.com

January 12th 2024

Health State Utility Scores for DMD-Related Impairment Need to Better Reflect Patient Input

Patient-reported outcomes data regarding measures of health-related quality of life, also known as health state utilities, in Duchenne muscular dystrophy (DMD) are scarce, with current knowledge in this area concentrated in caregiver feedback.

Sarepta Therapeutics Inc | Image Credit: Timon - stock.adobe.com

January 5th 2024

Sarepta Therapeutics Seeks to Expand Delandistrogene Moxeparvovec Indications

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

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

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

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.

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