Latest News
Isis Presents Update on Spinal Muscular Atrophy Drug Trials at the American Academy of Neurology Meeting
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 […]
Read More ›Tirasemtiv Demonstrates Improvements in Muscle Force, Grip Strength and Resistance to Fatigue in Preclinical Mouse Models of Spinal Muscular Atrophy
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 […]
Read More ›AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type I
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 […]
Read More ›Call Congress and Urge Passage of The ABLE Act
Congress appears to be on the verge of passing The Achieving a Better Life Experience (ABLE) Act of 2013, a bipartisan bill that would establish tax-exempt accounts to assist individuals […]
Read More ›Cure SMA Reaches Out to Newly Diagnosed Families
Cure SMA has invested nearly $57 million in SMA research in our 30-year history. But funding research is not the whole of our mission. We also provide compassionate, respectful support […]
Read More ›Isis Begins Phase III Clinical Trial
On August 1, Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The Phase III study, ENDEAR, […]
Read More ›