Latest News
Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
Scholar Rock today announced positive final top-line results from the Phase 1 clinical trial of its product candidate, SRK-015, a highly specific inhibitor of myostatin activation, in healthy adult volunteers. […]
Cure SMA to Host “Evening in the Park” at Disneyland Thanks to Support from AveXis
Conference attendees will enjoy a magical “Evening in the Park” at Disneyland thanks to special support from AveXis, a Novartis company. All registered attendees for the family conference will receive […]
AveXis and Cure SMA to Co-Host Webinar on the FDA’s Approval of Zolgensma
On Wednesday, June 12th, at 10am CDT (8am PDT/9am MDT/11am EDT) AveXis, a Novartis company, and Cure SMA will co-host a webinar on the FDA approval of Zolgensma. This webinar […]
AveXis Issues Community Statement on Zolgensma Approval
Dear SMA Community, We have exciting news to share with you — a major milestone has been reached in the fight against spinal muscular atrophy (SMA)! The US Food and […]
AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which […]
Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that […]