December 13, 2018
Bigoen has provided the below community statement on Spinraza.
Dear Members of the SMA community,
As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing...
December 12, 2018
AveXis has provided the following community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 - now known as ZOLGENSMA®.
Dear SMA Community,
We are providing this update to the SMA community in the U.S. at the request of Cure SMA....
December 3, 2018
Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA®...
November 29, 2018
Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific...
October 17, 2018
Collaborators Adrian Krainer, of Cold Spring Harbor Laboratory, and C. Frank Bennett, from Ionis Pharmaceuticals, received the 2019
October 8, 2018
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...
October 5, 2018
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...
October 3, 2018
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 and...
October 3, 2018
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...
September 12, 2018
The complexities of newborn screening require a sophisticated, multifaceted approach. With this in mind, Cure SMA has been working on newborn screening through several different avenues, bringing our full resources to ensure that babies born with SMA have the opportunity to receive early...