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 […]
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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
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 with disabilities and their families in saving private funds for the purpose of paying for a variety of qualified disability-related expenses. The legislation has very
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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 for all individuals and families who are affected by SMA. One of the primary ways we do this is through outreach to newly diagnosed families.
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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, is the first of several planned studies in a broad and comprehensive late-stage clinical development program for ISIS-SMNRx. Isis plans to initiate a second pivotal
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Cure SMA is proud to announce that we will be committing $1.8 million to new research funding over the next 12 months. Fifteen years ago, we had just one potential drug in the beginning stages of preclinical discovery. Today we have over a dozen, including five now in clinical trials. Though there’s great promise in
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