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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We are excited to announce that Families of SMA is now Cure SMA. In addition to this name change, we’ll also be making a number of other improvements to the way we communicate, starting with a new look and this new website. In 1984, a small group of families joined together so they could fund
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