Researchers report making a 10-fold improvement in a technology that permanently repairs flawed dystrophin genes, giving a real boost to this therapeutic strategy.
There are major shifts underway in understanding the physiological mechanisms that control muscle contraction, a field that has been the focus of intense research for centuries…
SECARS microscopy, a procedure which is based on quantum effects, with which certain molecules can be monitored virtually in real time, turns out to be a highly sensitive tool for the non-invasive research of the most varied diseases and may supplement the medical diagnosis tools of CT and MRI in the future…
ALS researchers are seeking participants for studies on the value of high-fat, high calorie diets; biological indicators called “biomarkers”; and the antibiotic ceftriaxone.
“Lumizyme has stopped the progression of my disease,” says one of first to receive the drug since the FDA granted commercial approval.
A study of exon-skipping drug GSK2402968 in nonambulatory subjects with DMD has opened at Nationwide Children’s Hospital in Ohio.
Local scientists, health advocates and leaders from the California Institute for Regenerative Medicine (CIRM) will gather in San Diego June 23 for a special public meeting to spotlight a new University of California, San Diego grant to pursue novel, fast-tracked stem cell-based therapies for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. The meeting, to be held at 8:30 a.m…
A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder.
The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered June 16 at the University of Florida. Pompe disease is a rare form of muscular dystrophy and has been the focus of a research program at UF for more than 10 years. It is now part of expanded efforts in neuromuscular disease research…
An experimental drug targets the immune system in ALS, switching cells from ‘attack’ to ‘protective’ mode.
