Recently, we announced that Dr. Lyndsay Murray at The University of Edinburgh will receive a a Cure SMA basic research grant of $80,000 over two years, to explore the reasons causing motor neuron cell death in SMA. Motor neurons are nerve cells in the brain stem and spinal cord that control muscle movement. We know […]
Cure SMA Announces Basic Research Funding for Dr. Lyndsay Murray Read More »
Cure SMA and Parent Project Muscular Dystrophy (PPMD) are conducting two different surveys about clinical trial participation. The goal is to find out more about how the neuromuscular community—individuals and families affected by conditions like spinal muscular atrophy or muscular dystrophy—thinks about potential therapeutics and clinical trials. All parents of children with SMA who live
At the end of last week, we announced the close of our for request for proposals (RFP) for clinical care research. A clinical care RFP is an invitation for scientists to submit their best ideas for projects that address the clinical, psychological or social aspects of SMA. They explain what they want to study, how
Funding Process Reaches Next Steps for Spinal Muscular Atrophy Clinical Care Research Read More »
Did you catch Cure SMA on Top Chef last week? Chef Takashi Yagihashi competed on behalf of Cure SMA for Bravo’s Top Chef Duels series. What a great opportunity to raise awareness of SMA, right on the heels of SMA Awareness Month! Chef Takashi and his family have been involved with Cure SMA for several
Chef Takashi Competes for Cure SMA Read More »
Cure SMA was featured in the July/August issue of Assay and Drug Development Technologies, a scientific journal that focuses on the development of new drugs. In honor of SMA Awareness Month, SMA was also featured on the front cover. Assay reaches scientists in 170 countries worldwide, making this a unique opportunity for us to influence
Cure SMA Featured in Assay and Drug Development Technologies Read More »
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 deficits in a mouse model of Spinal Muscular Atrophy (SMA). Scientists from Roche Pharma Research and Early Development (pRED), PTC Therapeutics, Inc., the SMA Foundation,
Spinal Muscular Atrophy Mice Model Results Published in the Journal Science Read More »
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
