Latest News
Cure SMA Announces New Subcommittee on Adults with SMA
Cure SMA recently announced the formation of the Subcommittee on Adult Outreach. This group will work to actively engage the adult SMA community in order to effectively support them. The […]
Read More ›Isis Releases Phase II Clinical Trial Results
Today, Isis released results from their Phase II open-label clinical trials of ISIS-SMNRx. These results covered two separate trials: a trial in infants with SMA type I, and a trial […]
Read More ›South Florida Gala of Hope Closes in on Milestone
On Thursday, October 2, Jennifer Miller Smith and Fiorenna Fuentes Israel, two dedicated mothers of children with SMA, planned an evening to be remembered in their quest to achieve a […]
Read More ›AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world’s first human gene therapy trial for the treatment of […]
Read More ›Isis Presents Phase II Trial Results
This Friday, October 10, Isis Pharmaceuticals will be presenting results from their Phase II clinical trials of ISIS-SMNRx in both children and infants. The results will be presented as part […]
Read More ›AveXis Receives Orphan Drug Designation
AveXis has received Orphan Drug Designation from the FDA for their gene therapy program, called chariSMA. Orphan Drug Designation is granted by the FDA to encourage and expedite the development […]
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