Research
Read the Fall 2014 Issue of Compass
The latest issue of Compass, our research publication, is now available online. This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and […]
Read More ›2014 SMA Researcher Meeting Summary: Clinical Trials
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: Therapy Development
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: SMA Pathology
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: The SMN Protein
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
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 […]
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 […]
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 […]
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 […]
Read More ›Isis Begins Recruiting for Phase III Trial
Isis is currently recruiting participants for Endear, a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The goal of this […]
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