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Isis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy
…M.D, Sidney Carter Professor of Neurology and Pediatrics and Co-Director of the Motor Neuron Center at Columbia University Medical Center. SMA is a severe genetic disease that affects approximately 30,000…
Read MoreTrophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy
…of funding.” More information about spinal muscular atrophy http://www.afm-telethon.fr http://www.curesma.org https://smafoundation.org/about-sma/ About Olesoxime Olesoxime (TRO19622) is the lead compound in Trophos’ proprietary cholesterol-oxime family of compounds that target and preserve…
Read MoreIsis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy
…M.D, Sidney Carter Professor of Neurology and Pediatrics and Co-Director of the Motor Neuron Center at Columbia University Medical Center. SMA is a severe genetic disease that affects approximately 30,000…
Read MoreIn Vitro Tools
…of a cell culture plate or test tube. In vitro assays are important because they provide the opportunity to test compounds and investigate SMA biology in human and animal model…
Read MoreBloomberg Article: New Hope for Kids With a Rare Disease, Thanks to a Dinner Party
…companies together at a dinner, which launched the deal that would speed nusinersen ahead. “This project is a prototypical example of how academia and industry and private foundations can” advance…
Read MoreSMA featured on the Charlie Rose Brain Series
…for the SMA Foundation’s Pediatric Neuromuscular Clinical Research (PNCR) Network. The show originally aired on Friday, July 20, 2012 and can now be viewed online at CharlieRose.com. [Photo Credit: www.charlierose.com]…
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