Spinal Muscular Atrophy (SMA)

Awesome Adventures


Reagan is MDA’s 2015 National Goodwill Ambassador.  Prior to that she served as the 2014 National Goodwill Ambassador and the Wisconsin State Goodwill Ambassador for three years.  Her favorite subjects are music, art and reading.  She began drawing at a very young age.  Reagan’s other “favorite” activity is dancing.

Reagan created this artwork to celebrate the 60th anniversary of MDA Summer Camp for youngsters affected with muscle diseases.

Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Type 2 and 3 SMA

News: Isis Pharmaceuticals Reports Data from Phase 2 Study in Infants with Spinal Muscular Atrophy

SMA: ISIS-SMNRx To Be Tested in Presymptomatic Infants

Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx  in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms. 

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

Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional studies could begin in the first half of 2015.

PTC To Test RG7800 in SMA

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.

AveXis SMA Gene Therapy Trial Continues Recruiting

Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a

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.