Our Forums moderator Kevin Schaefer published his list of entertainment options, perfect for these odd times we’re facing. To discuss, add more recommendations and leave your feedback please join the conversation in our Forums: click here. 1) If at Birth You Don’t Succeed by Zach Anner If…
Isis Pharmaceuticals recently announced it received $2.15 million from Biogen to advance CHERISH, the ongoing pivotal Phase 3 study assessing ISIS-SMNRx in children suffering with spinal muscular atrophy (SMA). Isis has now received over $120 million from Biogen to advance this investigational therapy. CHERISH a Phase 3 ISIS-SMNRx study; it is a double-blinded, randomized, 15 month study in almost 120…
Isis Pharmaceuticals, Inc.  recently revealed its latest data on the Phase 2, open-label and multiple-dose study of ISIS-SMNRx in children diagnosed with spinal muscular atrophy (SMA) who are still receiving treatment in an open-label extension (OLE) study. Muscle function scores and motor function tests were preformed in children receiving ISIS-SMNRx treatment, and data proved consistent with previous…
Francesco Lotti (https://www.curesma.org/news/grant-francesco-lotti.html) Francesco Lotti, PhD from Columbia University was recently awarded $140,000 in funding from Cure SMA to support the researcher’s current project, entitled “Role of Sumoylation in SMN Function and SMA Pathology.” SMN, which stands for “survival motor neuron,” is a protein crucial to the functioning of the nerves…
Dr. Barrington G. Burnett was named the recipient of the 2014 Emerging Investigator $62,500 Finalist Award, according to a recent Gwendolyn Strong Foundation and FightSMA announcement. Dr. Barrington G. Burnett’s project was entitled “Targeting the Ubiquitin Proteasome System to Treat Spinal Muscular Atrophy,” with the goal of identifying pathways and genes…
AveXis, Inc., a drug development company currently using gene therapy to address patients’ unmet needs suffering with orphan diseases such as Spinal Muscular Atrophy (SMA), has recently announced the completion of a $10 million financing set that was led by Deerfield Management and Roche Venture Fund. As part of the terms of…
Orphan drug development company AveXis Inc. recently announced the completion of the dosing phase in the low-dose cohort of the company’s ongoing clinical trial efforts for its experimental Type 1 spinal muscular atrophy (SMA) therapy. This is the first human gene therapy trial for the disease, which started enrolling participants in late April. SMA is the…
