Latest News
Team Cure SMA Celebrates Global Running Day!
Happy Global Running Day! Is Global Running Day a made-up holiday? Kind of. But unlike other made-up holidays dedicated to eating pancakes or taking selfies, Global Running Day has the […]
Read More ›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. […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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