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Roche, PTC & SMA Foundation Agreement to Develop a Treatment for SMA Receives Extensive Coverage
…Biology News, InPharm, MEDCITY Investing, NJBIZ, Pharma Times, PMLiVE Intelligence Online, QUEST – MDA’s Research and Health Magazine, RTT News, SCRIP, The Pharma Letter, The Pink Sheet Daily, and Xconomy….
Read MoreFrequently Asked Questions
…For more information about SMA including videos with medical experts, please visit the DNA Learning Center website (http://www.learnaboutsma.org/). What is the cause of SMA? Expand Unlike many other rare neuromuscular…
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 PHARMACEUTICALS REPORTS DATA FROM ISIS-SMNRX PHASE 2 STUDY IN INFANTS WITH SPINAL MUSCULAR ATROPHY
…Biogen to develop and potentially commercialize the investigational compound, ISIS-SMNRx, for the treatment of SMA. Under the terms of the January 2012 agreement, Isis is responsible for global development and…
Read MoreISIS PHARMACEUTICALS REPORTS INTERIM RESULTS FROM ISIS-SMNRX MULTIPLE DOSE STUDY IN CHILDREN WITH SPINAL MUSCULAR ATROPHY
…observed dose- and time-dependent increases in muscle function scores in children with SMA. The consistency of the results between the single-dose and the multiple-dose studies supports our earlier optimism around…
Read MoreIsis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy
…a devastating disease that leads to the loss of motor neurons resulting in muscle weakness and respiratory failure in children. The genetic cause of this disease is well understood, but…
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