August 2014

Originally published on April 29, 2014 The 66th American Academy of Neurology (AAN) Meeting is being held this week in Philadelphia. Over 10,000 neurologists are in attendance. Trophos presented late breaking data on their pivotal phase II/III trial on olesoxime in Spinal Muscular Atrophy there. Trophos is a clinical stage pharmaceutical company developing innovative therapeutics for

Originally published on April 29, 2014 Isis Pharmaceuticals, Inc. today provided an update on both of its ongoing open-label Phase 2 clinical studies of ISIS-SMNRx in infants and children with spinal muscular atrophy (SMA) at the 66th American Academy of Neurology (AAN) meeting in Philadelphia, PA.  Isis reports they are on track to initiate Phase 3

Originally published on June 26, 2014 Cytokinetics announced that data from preclinical research relating to tirasemtiv in mouse models of spinal muscular atrophy (SMA) were presented at the 2014 Annual Spinal Muscular Atrophy Conference in National Harbor, Maryland. In these models, tirasemtiv increased muscle force and improved grip strength, grid hang time, and resistance to

Originally published on June 26, 2014 AveXis, Inc., a synthetic biology platform company, announced today that the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type I has now been enrolled and dosed by Nationwide Children’s Hospital. This trial utilizes chariSMA, the gene therapy product developed by Dr. Brian Kaspar, of