Cure SMA recently granted a $304,000 drug discovery award to Livio Pellizzoni, assistant professor of Pathology & Cell Biology at Columbia University for a new research project with potential to treat spinal muscular atrophy (SMA). Pellizzoni and collaborators at Northwestern University have identified an altered cellular pathway in SMA patients, which may…
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Cure SMA granted just over $2 million in new research funding in 2015-16. The non-profit organization dedicated to advocacy and treatment research funding for spinal muscular atrophy (SMA), reports that the SMA drug development spectrum has expanded dramatically over the past decade, with six of the 18 programs currently in…
Cure SMA, a non-profit organization dedicated in large part to funding research for the treatment and cure of spinal muscular atrophy, reports that its drug development pipeline has grown dramatically over the past decade. Of 18 programs currently in the pipeline, six are in clinical trials, several of them…
Cure SMA has just released an update on its spinal muscular atrophy (SMA) drug pipeline and, according to a press release, the latest version now includes increased coverage of potential treatment approaches to SMA: 18 active programs, 14 pharmaceutical partners, six programs in clinical trials, and 28 programs…
Biogen and Ionis Pharmaceuticals have recently issued several updates on the clinical development of nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA). The partners announced that the enrollment period is now completed for both Phase 3 trials and for the Phase 2 study. Phase 3 trials remain on…
FightSMA, an all-volunteer, parent-led nonprofit, reported that the U.S. Senate Appropriations Committee has, as part of the Fiscal Year 2017 Defense Appropriations bill, included for a first time spinal muscular atrophy (SMA) as a research topic in the Defense Department’s Peer-Reviewed Medical Research Program. FightSMA calls this welcome…
Cure SMA recently announced that Christine DiDonato has been awarded a $70,000 research grant for her research study, “Assessing mediators of muscle weakness in SMA mice.” DiDonato is a basic science researcher who lives in Chicago and is currently working as an associate professor in the Department of Pediatrics…
AveXis presented positive pulmonary data from an interim analysis of its ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) type 1. The presentation, by Richard Shell, M.D., a member of the Section of Pulmonary Medicine at Nationwide Children’s Hospital, was made at the recent 2016 International Conference…
FightSMA has released a video from its Clinical Trial Panel, featuring a discussion among 10 physicians and scientists on the latest research developments in spinal muscular atrophy (SMA), that was part of its recent 2016 Annual Research Conference. The video release is the first of several planned from events at the conference,…
University of Missouri researchers have developed a gene replacement therapy for spinal muscular atrophy with respiratory distress type 1 (SMARD1). The therapy is able to cross the protective blood-brain barrier and target affected motor neurons in a non-invasive manner. The research paper describing the remarkable development, “Rescue of a…
