Duchenne Muscular Dystrophy

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.

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.

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.

The disease-modifying treatment landscape has expanded in recent years, but access to the latest approved Duchenne muscular dystrophy (DMD) treatments can be a challenge for patients and providers, Aravindhan Veerapandiyan, MD, a pediatric neuromuscular specialist at Arkansas Children’s Hospital, said.

Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior balance.

Aravindhan Veerapandiyan, MD, of the Division of Pediatric Neurology at the University of Arkansas for Medical Sciences, discussed the Duchenne muscular dystrophy (DMD) therapy landscape and barriers to treatment access.

Trials of Duchenne muscular dystrophy (DMD) treatments targeting declining lung or upper limb function, which are typically limited to nonambulatory patients, could also include ambulatory patients, according to the study's findings

Results from a national survey of patients with Duchenne muscular dystrophy (DMD) and their families found that patients and families want to be informed early about endocrine complications associated with glucocorticoid treatment.

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.

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).

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

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.

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).

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.

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.

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.

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.

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.

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).

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

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.

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).

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.

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.

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.

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.

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.

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

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.

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.