DMD News Briefs

statistical graphs

Mortality Rates in DMD Vary by Race and Treatment Regimen

The median estimated age of death in patients with Duchenne muscular dystrophy (DMD) is just under 24 years, with elevated mortality rates in non-Hispanic Black patients compared to non-Hispanic White patients, according to a study recently published in Neuromuscular Disorders. Since 2004, the study has analyzed and longitudinally followed 526 male patients born between 1982…

chromosome DNA

First US Sites Open to Restart Phase 3 DMD Gene Therapy Trial

The first US clinical sites will be opening to restart Pfizer’s Duchenne muscular dystrophy (DMD) gene therapy trial, as published in the company’s press release. This is following the lifting of the trial’s hold by the US Food and Drug Administration (FDA). The global randomized, placebo-controlled phase 3 clinical trial, called CIFFREO, (NCT04281485) is investigating…

home technology

New Home-Based Tool Expands Assessment Options for DMD

The development of scorecards to evaluate a video assessment system for Duchenne muscular dystrophy (DMD) was described and published in the PLOS One and was then announced in a press release by Casimir, the developer of the system called Duchenne Video Assessment (DVA). Physical therapists experienced in evaluating patients with DMD “confirmed that the compensatory…

microtiter plate

Safety and Functional Improvements Found With Chimeric Cell Therapy for DMD

Dystrogen Therapeutics announced positive results for patients with Duchenne muscular dystrophy (DMD) from an ongoing investigation in Poland as part of the dystrophin expressing chimeric (DEC) protocol known as DT-DEC01. The trial assessed 1-3 month functional results and safety data in a low-dose cohort, in which no adverse events were observed. The treatment engrafts skeletal…

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Low-Level Laser Therapy With Exercise Improves Utrophin Levels in DMD

Researchers revealed extra benefits from the combination of low-level laser therapy (LLLT) with exercise in a mouse model of Duchenne muscular dystrophy (DMD) and published their results in Lasers in Medical Science. The researchers found increases in muscle strength and utrophin levels and reductions in muscle degeneration and inflammation as a result of the combined…

gene research

Certain Mesenchymal Stem Cells Could Have Therapeutic Potential in DMD

Mesenchymal stem cells derived from Wharton’s jelly expressing aurora kinase A (AURKA) could have therapeutic effects in patients with Duchenne muscular dystrophy (DMD), according to a new study published in Stem Cells International.  Research has shown that many proteins secreted by mesenchymal stem cells may have therapeutic effects in DMD and other diseases. Therefore, mesenchymal…

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