Patricia Inácio, PhD, science writer —

The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from the MoveDMD trial report. Specifically, edasalonexent — administered orally as 100 mg/kg dose for 48 weeks — resulted in a statistically significant delay in disease progression and signs of…

Magnetic nanotubes with a spear-like tip may enhance the precision and effectiveness of gene therapy delivery, a promising therapeutic strategy for many genetic diseases, including muscular dystrophy, according to UCLA researchers. The study, “Precision-Guided Nanospears for Targeted and High-Throughput Intracellular Gene Delivery,” was published in the journal ACS Nano.

Scientists are increasingly looking at gene therapies as potential treatments for Duchenne muscular dystrophy. But there are a lot of ways to do them, and an expert in the field shed light on the subject in a webinar arranged by Parent Project Muscular Dystrophy (PPMD). Dr. Timothy Cripe discussed the…

Summit Therapeutics has opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy (DMD) who participated in the previous Phase 1 clinical trials for the investigational therapy ezutromid (SMT C1100) but who, at the time of enrollment, failed to meet the enrollment…

A new interim analysis of the PhaseOut DMD clinical trial shows that ezutromid (SMT C1100) significantly decreased muscle inflammation in patients with Duchenne muscular dystrophy (DMD), Summit Therapeutics announced. Ezutromid is called a utrophin modulator because it is able to trigger utrophin production in people who lack dystrophin,…

PTC Therapeutics is recruiting Duchenne muscular dystrophy (DMD) patients ages 5 and older and with a nonsense mutation for a long-term Phase 3 study to characterize the effects of Translarna (ataluren) on disease progression. The trial (NCT03179631) opened in July 2017 and enrolling patients at eight sites across…

Measuring the physical strength of individual cells is now 100 times faster with a new device called FLECS developed by researchers at UCLA and Rutgers University. FLECS allows researchers to measure the strength of thousands of individual cells at a time and could make it easier for them…

European and Japanese researchers used an artificial chromosome to generate the dystrophin protein that’s missing in Duchenne muscular dystrophy. The chromosome delivered a two-gene package capable of producing the missing protein to a mouse model of the disease. The team also used it in a lab setting involving human cells.

Researchers have found that cognitive function, including the ability to multitask and solve problems, may be impaired in boys with Duchenne muscular dystrophy (DMD) — even in those with normal and above average IQs. Their study, “Cognitive profile in Duchenne muscular dystrophy boys without intellectual disability: The role of executive functions,”…

The first Duchenne muscular dystrophy (DMD) patient has received systemic microdystrophin gene therapy as part of a Phase 1/2 clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The trial (NCT03375164), currently recruiting participants, will test the safety and efficacy of a single dose of intravenous microdystrophin gene therapy…