Duchenne Muscular Dystrophy

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.

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).

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.

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).

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

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.

The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).

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.

Septal circumferential strain (Ecc) was significantly decreased in LGE-negative boys with Duchenne muscular dystrophy (DMD) and correlated to ventricle changes.

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.

From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and research.

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.

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).

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.

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

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.