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.
Cure SMA Reaches Out to Newly Diagnosed Families Read More »
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
Cure SMA Announces New Research Funding Read More »
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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