Author Archives: Ana Pamplona, PhD

Researchers say it is important to carefully assess the timing of the start of corticosteroid treatment for Duchenne muscular dystrophy (DMD) in children and its potential association with disease outcomes. The study, “Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy,” was published in the…

A novel study from Max Planck Institute for Heart and Lung Research investigators revealed a novel protein, Prmt5, as a crucial player in the regulation of muscle stem cells during muscle regeneration. The study entitled “Prmt5 is a regulator of muscle stem cell expansion in adult mice”…

A late-stage clinical trial performed by researchers at the University of Florida Health (UF Health) led to a new promising treatment for children with the most common form of muscular dystrophy, Duchenne Muscular Dystrophy. This condition causes the loss of mobility when the patients reach 12 years old, and…

BioBlast Pharma Ltd. presented the preliminary results from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Occulopharyngeal Muscular Dystrophy (OPMD), a progressive, incapacitating genetic disease. Cabaletta is a chemical chaperone that has been shown to reduce pathological aggregation of proteins within cells in several diseases…

Insights into recent advances for Muscular dystrophy disease treatment, entitled “HDAC-regulated myomiRs control BAF60 variant exchange and direct the functional phenotype of fibro-adipogenic progenitors in dystrophic muscles” were recently published in the Genes & Development journal by Dr. Valentina Saccone. Dr. Saccone is part of Dr. Pier Lorenzo Puri’s…