Front Page 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 […]
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 […]
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 […]
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 […]
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 […]
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. […]
Read More ›Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA
Cytokinetics, Incorporated and Astellas Pharma Inc. today provided an update for their collaborative skeletal muscle program, including clinical trial updates for reldesemtiv in SMA. In […]
Read More ›Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH […]
Read More ›Genentech Releases Community Statement on Risdiplam
The following statement is a community update from Genentech/Roche on the risdiplam studies FIREFISH and SUNFISH. Dear SMA community, We are happy to share an […]
Read More ›Community Spotlight: Adrienne Vollmer
In May, Governor Eric Holcomb held a signing ceremony for Indiana House Bill 1017, adopting newborn screening for SMA and SCID. Dubbed “Graham’s Bill” in […]
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