Saturday morning’s presentations were devoted to the latest news about clinical trials and drug development. Among the highlights: Duchenne muscular dystrophy (DMD): Edward Kaye from Sarepta Therapeutics presented his company’s most recent data about its experimental drug eteplirsen, designed to treat about 13 percent of the Duchenne muscular dystrophy (DMD) population, and discussed plans for . . .
Friday’s afternoon sessions focused on laboratory studies in animal and cell models of neuromuscular diseases. These kinds of studies are necessary before treatments can be tested in humans. Among the highlights: Spinal muscular atrophy (SMA): Brian Kaspar from Nationwide Children’s Hospital in Columbus, Ohio, presented experiments from his lab to develop gene transfer therapy for . . .
Friday’s morning sessions focused on deepening understanding of the mechanisms underlying diseases of the muscles and nervous system Among the highlights: Spinal muscular atrophy (SMA): Christine DiDonato from Northwestern University presented her work with mice that have a disease mimicking spinal muscular atrophy (SMA) with varying degrees of severity, which is characteristic of human SMA . . .
Thursday’s afternoon sessions focused on therapeutic approaches now in development Here are some highlights: Duchenne muscular dystrophy (DMD): Dean Burkin from the University of Nevada, Reno, presented his team’s findings that show how raising levels of a protein called alpha 7 beta 1 integrin has potential as a therapy for treating Duchenne muscular dystrophy. A . . .
Thursday morning focused on understanding the genetics and the “epigenetics” of neuromuscular disorders Epigenetics is the relatively new field of factors that influence the activity of genes but do not change the basic coding (DNA sequence) of a gene. Identifying the genetic and epigenetic pathways of a disease can allow for better diagnosis and genetic . . .
CHICAGO, Feb. 27, 2015 – Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA’s . . .
I recently stumbled upon a definition of the word “rare” that profoundly unnerved me: “rare — not found in large numbers and consequently not of interest or value.” As a neurologist for nearly three decades, my colleagues and I in the medical field consider a disease or disorder to be “rare” in the United States . . .
Product holds potential for replacing glucocorticoids in Duchenne muscular dystrophy and other chronic inflammatory states SILVER SPRING, M.D./ CHICAGO – February 18, 2015 – ReveraGen BioPharma today announced the start of a Phase 1, first-in-human, clinical trial of a novel dissociative steroidal drug, VBP15, under development for Duchenne muscular dystrophy. Recruitment in the trial is . . .
Dear ALS Community, We are excited and enthusiastic about the new approaches and new drugs that are being tested for people with ALS and remain very hopeful that one or more of these will become a therapy with substantial impact on slowing down or perhaps even stopping ALS. We remain grateful to the brave volunteers . . .