Patricia Inácio, PhD,  science writer—

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…

Capricor Therapeutics is expanding its Duchenne muscular dystrophy (DMD) and cardiac therapy programs using cardiosphere-derived cells (CDCs) by adding seven new patent applications related to the technology. The new applications were from Capricor’s existing exclusive license agreements with Cedars-Sinai Medical Center, and are aimed at expanding Capricor’s cellular and…

Scientists at King’s College London, UK discovered that the PAX7 gene and its regulatory gene network is a new hallmark of facioscapulohumeral muscular dystrophy (FSHD). Moreover, PAX7 works better as a biomarker for the disease than the current leading candidate gene…

Researchers have found that lowering the the levels of a protein called sarcolipin lessens muscle weakness and improves skeletal and cardiac muscle function in a mouse model of Duchenne muscular dystrophy (DMD). These findings suggest that therapeutics targeting sarcolipin may have significant benefits to DMD patients. The study “Reducing sarcolipin expression…

Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 1/2 trial, called IGNITE DMD, will soon open for patient enrollment in the…