Spinal Muscular Atrophy (SMA)

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

PTC To Test RG7800 in SMA

AveXis SMA Gene Therapy Trial Continues Recruiting

SMA - Lyndsay Murray, Ph.D.

Lyndsay Murray, a lecturer in anatomy at  the University of Edinburgh in Scotland, has been awarded an MDA development grant totaling $152,280 over three years to determine the earliest changes in gene activity that occur in spinal muscular atrophy (SMA). By conducting experiments in mice with and without an SMA-like disorder, Murray and colleagues will study the genetic changes that occur prior to the death of nerve cells in the SMA-like condition.

SMA - Rashmi Kothary, Ph.D.

Rashmi Kothary, a senior scientist in the Regenerative Medicine Program at Ottawa Hospital Research Institute in Canada, has been awarded an MDA research grant totaling $253,800 over three years to further investigate a potential new treatment approach for spinal muscular atrophy (SMA) . Kothary and colleagues will continue to develop inhibitors of an enzyme called rho kinase to see if they are beneficial in this disease. Early experiments in mice have shown promise.

SMA - Stephen Kolb, M.D., Ph.D.

Stephen Kolb, an assistant professor in the Departments of Neurology and of Molecular & Cellular Biochemistry at Ohio State University, has been awarded an MDA human clinical trial grant totaling $183,354 over three years as supplemental funding for the SMA NeuroNEXT biomarkers study. This study is being conducted by the U.S. National Institutes of Health (NIH) to compare children with and without spinal muscular atrophy (SMA) during the first two years of life.

SMA: Further Data on ISIS-SMNRx Shows Promising Results

Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:

ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA

First Human SMA Gene Transfer Therapy Trial Opens

SMA: ISIS-SMNRx Shows Benefit in Infants, Children