Research
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 […]
Read More ›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 […]
Read More ›Catch Up on Research News from our Family Friendly Poster Session
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in […]
Read More ›Spinal Muscular Atrophy Biomarker Study Reaches Enrollment Goals
We’re excited to announce that enrollment for the NeuroNEXT biomarker study is now fully filled. This means that this critical study can continue to progress […]
Read More ›RFP Closes for Spinal Muscular Atrophy Basic Research Funding
On September 5, we announced the close of our for request for proposals (RFP) for basic research. An RFP is an invitation for scientists to submit […]
Read More ›Cure SMA Announces Basic Research Funding for Dr. Lyndsay Murray
Recently, we announced that Dr. Lyndsay Murray at The University of Edinburgh will receive a a Cure SMA basic research grant of $80,000 over two […]
Read More ›Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
Cure SMA and Parent Project Muscular Dystrophy (PPMD) are conducting two different surveys about clinical trial participation. The goal is to find out more about […]
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