Research
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. […]
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. […]
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. […]
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. […]
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 […]
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 […]
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 […]
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 […]
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 […]
Cure SMA-Funded CALIBR Research Collaboration Expanded to USC
Cure SMA is pleased to announce that we have expanded our research collaboration with the California Institute for Biomedical Research (CALIBR) to include a collaborative […]