Duchenne Muscular Dystrophy

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.

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.

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.

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.

A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).

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

On this episode of Managed Care Cast, we're talking with the author of a study published in the July 2024 issue of The American Journal of Managed Care® that estimates the insurance value of novel Duchenne muscular dystrophy (DMD) treatment.

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.

Migvis Monduy, MD, medical director of Neuromuscular and Movement Disorders Programs at Nicklaus Children's Hospital, discussed the most promising areas of research in Duchenne muscular dystrophy (DMD), as well as the mechanisms of gene therapies for DMD.

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.