Latest News
AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I
AveXis, Inc., a Novartis company, today announced that they have filed for FDA approval of AVXS-101, a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is […]
Read More ›North Carolina to Screen Newborns for SMA Through Early Check
Early Check, a new research study led by RTI International, is now available for newborn babies in North Carolina. Early Check is a free screening study designed to identify children […]
Read More ›Breakthrough Prize Awarded to Adrian Krainer and Frank Bennett for SMA Research Leading to Spinraza
Collaborators Adrian Krainer, of Cold Spring Harbor Laboratory, and C. Frank Bennett, from Ionis Pharmaceuticals, received the 2019 Breakthrough Prize in Life Sciences. They won for the development of the […]
Read More ›Cure SMA Receives Generous Funding from Luke 18:1 Foundation
Cure SMA would like to thank the Luke 18:1 Foundation for their generous donations to our equipment pool and research programs. The foundation’s generosity will provide support and hope to […]
Read More ›SMA Care Center Network and Clinical Data Registry Launched
Cure SMA today announced the launch of our SMA Care Center Network. The SMA Care Center Network is the centerpiece of our efforts to address the changing landscape of SMA. […]
Read More ›New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA
Biogen, Inc. announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25 presymptomatic infants with SMA. The data were presented Sunday […]
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