Long-term use of steroid treatment is linked to increased weight gain in boys with Duchenne muscular dystrophy (DMD) who are unable to walk, a study shows. Recognition of the effect of steroid therapy on patients’ weight and growth progression can have important implications on treatment management and early diagnosis of…
Flagship Biosciences has developed an automated method to analyze muscle fibers (myofibers) and quantify key disease-related biomarkers, such as dystrophin, for Duchenne muscular dystrophy (DMD). The algorithm, known as MuscleMap, was designed to screen muscle tissue samples and detect fluorescent dyes that tag with increased precision the protein…
Catabasis Pharmaceuticals is launching a global Phase 3 trial to evaluate the potential of edasalonexent to treat boys with Duchenne muscular dystrophy (DMD) regardless of mutation type. The soon-to-open PolarisDMD trial is a global and one-year, placebo-controlled study that will assess the safety and efficacy of edasalonexent in boys, ages 4 through…
The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD), the company announced in a press release. On July 25, the FDA placed the…
There are many skills to learn if you recently have been diagnosed with a chronic illness. These range from being organized for doctor’s appointments to finding the best mobility assistance equipment. In today’s column, I will share my experience choosing a power wheelchair. I got my current…
On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach To Cure MD initiative during college football games. The fundraising effort, in its 11th consecutive year, has raised almost $2 million to fight Duchenne muscular dystrophy (DMD) and has…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type 2A (LGMD2A). LGMD2A, also known as calpainopathy, is the most common subtype of limb girdle muscular dystrophy (LGMD),…
Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial…
One of the best days of my life was when my family and I brought home my dog, Andre. The second my eyes fell on him, I melted into a puddle, overwhelmed by his cuteness. He was the most adorable ball of fluff and I couldn’t fathom that…
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