Duchenne Muscular Dystrophy

With improved survival becoming more common among individuals who have Duchenne muscular dystrophy (DMD), cardiomyopathy is also increasing in prevalence among this population.

This retrospective review compared appendicular lean mass (ALM) and ALM index among patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.

This comprehensive comparative analysis examined the economic and health care resource utilization implications of initiating glucocorticoid and exon-skipping therapy for Duchenne muscular dystrophy (DMD).

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.

In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.

Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.

A new study looks at the balancing act physicians and families must carry out when managing expectations at a time of great scientific advancement in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

Researchers provided an overview of what they deem a hopeful future for spinal muscular atrophy and Duchenne muscular dystrophy.

Studies with better data are still needed to fully understand the epidemiology of Duchenne muscular dystrophy (DMD), according to a recently published review.