Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication.
Sep 19, 2016
0
1
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
"When you hear hoofbeats, think horses, not zebras," beginning med students learn. Ultrarare diseases are more like unicorns.
Aug 22, 2016
0
5
Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug ...
Jun 1, 2016
0
40
(HealthDay)—The drug eteplirsen should not be approved for treatment of Duchenne muscular dystrophy, a U.S. Food and Drug Administration advisory panel said Monday.
Apr 27, 2016
0
3
Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper published in The Journal ...
Apr 18, 2016
0
5
In a new study, a research team at Basel University Hospital in Switzerland investigates the biochemical and physiological characteristics of orbicularis oculi, a group of facial muscles that control the eyelids and are selectively ...
Apr 11, 2016
0
13
At a time when breakthrough therapies for certain cancers, hepatitis C, and rare diseases remain out of reach for many patients due to their prohibitive cost, new research by Dana-Farber Cancer Institute and MIT Sloan School ...
Feb 24, 2016
0
50
Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne ...
Feb 12, 2016
0
57
