Isis Pharmaceuticals will test its experimental antisense drug in 24 children with spinal muscular atrophy at five U.S. centers
posted on January 2, 2012 - 6:00am
A 24-person, phase 1 trial to test the safety and tolerability of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at Columbia University Medical Center in New York, with additional sites expected to open in Boston, Philadelphia, Dallas and Salt Lake City.
posted on January 1, 2012 - 1:42pm
Stem cells have been much in the news lately, including for neuromuscular diseases.
One extremely promising approach has been to create stem cells from the adult (differentiated) cells of humans or animals, and then reprogram them back to a stemlike state, after which they can be redifferentiated into a desired cell type, such as muscle or nerve cells. Stem cells made from differentiated cells...
A collaboration between PTC Therapeutics and Swiss biotech Roche may speed development of experimental treatments for spinal muscular atrophy
posted on December 1, 2011 - 6:00am
Swiss biotechnology company Roche, and PTC Therapeutics in South Plainfield, N.J., on Nov. 29, 2011, announced a collaboration through which the two companies will work together to advance drug development for spinal muscular atrophy (SMA).
Experiments in SMA mice suggest that antisense treatment is most effective when it reaches all body tissues, not just the central nervous system
posted on October 4, 2011 - 2:11pm
Treatment of a mouse model of severe spinal muscular atrophy (SMA) with an antisense oligonucleotide results in greater and longer-lasting benefit when given systemically than when given only to the central nervous system, new research shows.
Reversing muscle damage caused by low SMN; nerve-muscle communication breakdown; phase 2 trial of olesoxime now fully enrolled
posted on September 27, 2011 - 4:35pm
Skeletal muscle damage reversed in SMA mice
In addition to muscle damage caused by the loss of motor neurons in the spinal cord, skeletal muscle degeneration in spinal muscular atrophy (SMA) also stems from low levels of the protein SMN in muscle fibers. Now a team of scientists from Germany and the United Kingdom reports that muscle damage caused by low SMN can be reversed with drugs.
Several recent studies have found that the stem cell 'reprogramming' processes can lead to dangerous abnormalities
posted on September 20, 2011 - 10:52am
Stem cells have been much in the news lately, including for neuromuscular diseases.
Participants will be randomly assigned to a strength or stretching exercise program, and undergo multiple evaluations
posted on August 9, 2011 - 12:00am
The National Institute of Neurological Disorders and Stroke (NINDS), part of the U.S. National Institutes of Health (NIH), is conducting a study to assess the safety and effectiveness of exercise in men with spinal-bulbar muscular atrophy (SBMA), also known as Kennedy disease.
The utility of exercise in this disease is uncertain.
