Research
Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101
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® […]
Read More ›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 […]
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 ›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 ›Cure SMA’s Approach to Newborn Screening
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 […]
Read More ›Cure SMA Provides Funding for “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Published in Neurology
In a recent paper titled, “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Dr. Chad Heatwole and others describe a cross-sectional study of […]
Read More ›Updated 2018 SMA Standards of Care Statements Available Online
The 2018 SMA Standards of Care recommendations were published in Neuromuscular Disorders in February 2018 and March 2018 and are available online through Open Access […]
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