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Spinal Muscular Atrophy Mice Model Results Published in the Journal Science
On August 7, the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth prevents […]
Read More ›Trophos Presents Pivotal Phase II/III Study Results on Olesoxime in Spinal Muscular Atrophy at The American Academy of Neurology Meeting
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 […]
Read More ›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 […]
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