The SMA Project, a National Institutes of Health program supported by SMA patient advocacy groups, reports on its therapy development strategy in spinal muscular atrophy
A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the National Institute of Neurological Disorders and Stroke (NINDS) to accelerate the development of therapeutic candidates for thi
MDA will sponsor four symposia addressing specific roadblocks and moving forward with therapy development for neuromuscular disease
This month, MDA is launching a new U.S.-based symposium series to address important topics and ultimately to accelerate therapy development for neuromuscular diseases.
"MDA’s new symposium series is designed to address cutting-edge issues in neuromuscular disease research," said Sanjay Bidichandani, MDA's vice president of research. "Science is advancing at a rapid pace, and these small and...
Wake Forest researcher receives $369,365 for preclinical testing of gene transfer therapy for myotubular myopathy
MDA’s translational research program has announced it is funding research into a potential treatment for the inherited muscle disorder X-linked myotubular myopathy (MTM).
The grant of $369,365 grant to Wake Forest University professor Martin Childers will fund a three-year study of myotubularin gene transfer in mice and dogs that have an MTM-like disease.
MDA's research program in 2010
As MDA's research program heads into 2010, new directions, strategies and partnerships are under way.
All have the ultimate goal of moving research through the “pipeline” from basic science discoveries in university laboratories, to biotechnology development, and ultimately into viable therapies that can be prescribed in the clinic.
Below are examples of how this “pipeline” research approach is...
What can laboratory studies tell us about human disease?
This spring, the Duchenne muscular dystrophy community rejoiced when PTC Therapeutics of South Plainfield, N.J., announced that an experimental drug aimed at the disease had passed safety tests in human volunteers and would now be tested in boys with DMD.
This drug, like many others, was successfully tested in mice lacking dystrophin, the protein that’s also missing in the human form of the...
In this article: The latest news on clinical trials and studies related to Duchenne MD, spinal muscular atrophy, McArdle's disease, myotonic dystrophy, myasthenia gravis, Pompe's disease, diagnosis, translational research
New technology could propel rapid drug discovery for DMD and SMA
In the mid-1980s, when MDA-supported researchers began searching for the defective gene underlying Duchenne muscular dystrophy (DMD), the scientific community at large was pessimistic about the outcome.