Solid Biosciences Founder Ilan Ganot Discusses His Quest for a Cure for DMD
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ilan Ganot, founder and CEO of Solid Biosciences, based in Cambridge, Massachusetts.
DMD News Briefs
An Interview With DMD Expert and Gene Therapy Pioneer Dr. Jerry Mendell
Rare Disease Advisor senior correspondent Larry Luxner interviews DMD expert Jerry Mendell, MD, a professor at Nationwide Children’s Hospital in Columbus, Ohio.
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…
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…
CureDuchenne Holding National DMD Conference in May 2022
CureDuchenne’s national FUTURES conference on DMD will be held between May 27, 2022, and May 29, 2022, in Orlando, Florida, and virtually.
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…
Patient Advocate Laura McLinn Discusses Her Son’s Journey With DMD
Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Laura McLinn, the Indiana mom of 12-year-old Jordan McLinn, who has Duchenne muscular dystrophy (DMD). Jordan, who became the unofficial poster boy of the “Right to Try” movement, has just completed 5 years in a clinical trial for a novel exon 53 skipping therapy. Read…
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…
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…
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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