Junction: Duchenne Muscular Dystrophy

Astrocytes in Duchenne Muscular Dystrophy May Be Therapeutic Targets

The function of astrocytes is altered in Duchenne muscular dystrophy (DMD), according to a new study published in Glia. Jenny Lange, PhD, and the coauthors of the study suggest that defective astrocyte responses may contribute to neural impairment in patients with DMD. The data also suggest that targeting astrocytes could be a viable therapeutic option…

Ataluren Delays Respiratory Decline and Loss of Ambulation in Patients With Nonsense Mutation DMD

A long-term phase 3 study found that ataluren plus standard of care (SoC) delays progression of nonsense mutation Duchenne muscular dystrophy (nmDMD) and benefits ambulatory and nonambulatory patients. Ataluren is an oral therapy for patients with nmDMD that enables ribosomes to read through a premature stop codon in mRNA, allowing translation to continue and resulting…

Resolvin-D2 May Have Therapeutic Potential for DMD

Resolvin-D2 (RvD2) showed more potent effects than glucocorticoids in reducing inflammation and fibrosis in mice with Duchenne muscular dystrophy (DMD), according to a new study published in Nature Communications. The authors showed that treatment with RvD2 led to changes in macrophage phenotype, from proinflammatory to anti-inflammatory, and increased the secretion of promyogenic factors. These factors…

Wheelchair Hockey Can Improve Well-Being for Patients With SMA or DMD

Patients with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) who participated in wheelchair hockey had higher physical and psychological well-being scores than patients who did not participate in sports, according to a new Italian study. The results, published in the journal PM&R, found higher scores on the Quality of Life Index (QLI) as…

Life Expectancy, Survival Rates Improving Among Patients With DMD

Life expectancy is improving among individuals with Duchenne muscular dystrophy (DMD), according to a meta-analysis published in Neurology. Study results showed that patients with DMD born following 1990 had an increased median life expectancy of 28.1 years, compared with a combined cohort median life expectancy of 22.0 years. The study provides comprehensive survival probabilities and…

Creatine Kinase Test Shows Promise for Duchenne Muscular Dystrophy Screening

The creatine kinase (CK) test demonstrated good accuracy in screening for cases of Duchenne muscular dystrophy (DMD) in newborns, a systematic review found. The results of the review, which included 11 studies and a total of 1,416,123 newborns, were recently published in the journal Clinical Biochemistry. “Our review demonstrated a considerably low number of DMD…

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