A multinational pharmaceutical company has committed to developing Prosensa's exon skipping compound for DMD
On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon skipping” compound PRO051 for Duchenne muscular dystrophy (DMD).
The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.
Scientists use a small molecule to coax synthesis of a needed protein in SMA-affected cells
Scientists at three U.S. institutions have used a very small synthetic molecule to correct the genetic defect in cells taken from a person with spinal muscular atrophy (SMA), a disease in which muscle-controlling nerve cells in the spinal cord are lost.
Researchers add support to evidence that the blood-spinal cord barrier is abnormally leaky in ALS
Proteins that keep large molecules from moving freely across blood-vessel walls in the spinal cord appear to be deficient in people with ALS (amyotrophic lateral sclerosis), MDA-supported researchers say. They don't yet know, however, whether a lower-than-normal level of some of these so-called "tight junction" proteins, is helpful or harmful in the disease process.