For the first time, scientists found that in spinal muscular atrophy (SMA), the affected nerve cells that control muscle movement, or motor neurons, have defects in their mitochondria, which generate energy used by the cell. ...
Sep 15, 2016
0
18
Duchenne muscular dystrophy is a chronic disease causing severe muscle degeneration that is ultimately fatal. As the disease progresses, muscle precursor cells lose the ability to create new musclar tissue, leading to faster ...
Sep 12, 2016
0
2
New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings, which appear this month in the journal Neurology, could pave the way for first U.S.-approved ...
Sep 9, 2016
0
0
If a disease affects motoneurons, cells that control voluntary muscle activity, researchers should focus their efforts on motoneurons to find potential treatments, right?
Aug 31, 2016
0
0
"When you hear hoofbeats, think horses, not zebras," beginning med students learn. Ultrarare diseases are more like unicorns.
Aug 22, 2016
0
5
Researchers at Iowa State University have discovered what could lead to a new treatment for spinal muscular atrophy, a potentially fatal genetic disorder in young children.
Aug 9, 2016
0
106
In an announcement that delighted the families of patients and surprised many pharmaceutical industry analysts, the biotechnology firms Biogen and Ionis Pharmaceuticals on Monday said they had decided to cut short a phase-3 ...
Aug 4, 2016
0
6
A new study by a team of scientists from the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) could help develop treatment strategies for a crippling disorder ...
Aug 1, 2016
0
4
A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease.
Jul 27, 2016
1
134
According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over ...
Jul 25, 2016
0
0
