AveXis has provided the following community statement on their FDA filing for SMA type I. Dear SMA Community, AveXis, a Novartis company, is pleased to let you know that we have submitted regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important and […]
AveXis Issues Community Statement on FDA Filing for SMA Type I Read More »
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 missing or mutated in individuals with SMA. This first filing is for intravenous (IV) delivery of gene therapy. Based on typical practices, the expectation is
AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I Read More »
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 with rare health conditions before symptoms appear and study the benefits of early treatments. New and expectant mothers may enroll in the program online from
North Carolina to Screen Newborns for SMA Through Early Check Read More »
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 first effective therapy for spinal muscular atrophy, Spinraza. Dr. Krainer is a long time member of the the CURE SMA Scientific Advisory Board, and his
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 all those affected by SMA. The Luke 18:1 Foundation was started by Nicole and Daniel Stickane in honor of their son Luke who was diagnosed
Cure SMA Receives Generous Funding from Luke 18:1 Foundation Read More »
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. The goal of the SMA Care Center Network is to develop an evidence-based standard of care that will improve the lives of all those affected
SMA Care Center Network and Clinical Data Registry Launched Read More »
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 in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina. The interim analysis evaluated survival and
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 June, Cytokinetics announced data from a Phase 2 double-blind, randomized, placebo-controlled clinical study in patients with SMA which was designed to determine potential pharmacodynamic effects of a suspension formulation of
Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA Read More »
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 and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). In the FIREFISH study in Type 1 SMA, six out of 14 infants (43 percent) were able to sit
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 update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support and partnership of SMA Patient Groups around the world and to thank
Genentech Releases Community Statement on Risdiplam Read More »
