Iqra Mumal, MSc,  —

Researchers have found that skeletal muscle magnetic resonance imaging (MRI) correlates with motor function and can help predict the degree of functional deterioration in patients with Becker muscular dystrophy (BMD). A study with that finding, “Muscle MRI and functional outcome measures in Becker muscular dystrophy,” was published in the…

In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy. Researchers have now discovered that utrophin can reduce damage to energy-producing cell components known as mitochondria, whose deterioration is linked to MD. The reduction in damage improved the muscle…

Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy. Genes consists of dozens of components called exons. If an exon is missing, a gene can fail to produce a protein the body needs.

Sarepta Therapeutics is working with Duke University’s biomedical engineering program program on gene editing therapies for Duchenne muscular dystrophy. The editing approach the partners are using is called CRISPR/Cas9. It can correct mutations by removing regions of a gene that contain abnormal material. Removing the region restores the function of the protein that…

Researchers have found that reducing levels of elevated sarcolipin protein may be a potentially effective therapeutic strategy in the treatment of Duchenne muscular dystrophy (DMD) patients. The study, “Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice,” was published in Nature Communications. Current therapies…

Tricyclo-DNA (tcDNA), a class of therapeutics known as antisense oligonucleotides (AONs), has shown an important degree of effectiveness and a promising safety profile in a preclinical study with mice. The goal of the study is to test tcDNA’s potential use in treating Duchenne Muscular Dystrophy (DMD). The study, “Efficacy and…

A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed. Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and…

The drug discovery and development biopharma Summit Therapeutics is giving several presentations related to the ongoing PhaseOut DMD clinical trial at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. One of the poster presentations includes baseline…

In what could be the first step on the long road to finding a potential cure, researchers have found that aberrations in RNA processing are the major cause of congenital myotonic dystrophy (CDM). The study “Disrupted prenatal RNA processing and myogenesis in…

Researchers have found Duchenne muscular dystrophy (DMD) patients with heart disease have very high levels of ST2, an interleukin 1 receptor-like 1 protein. That makes this molecule a potential biomarker for cardiac disease in such patients. The study by scientists at Children’s National Health System in Washington, D.C., “Interleukin 1…