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 . . .
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 . . .