News

AveXis, Inc., announced that its Phase 1 clinical trial of its proprietary gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1 has now completed patient enrollment. Fifteen young patients, diagnosed before age six months and with two copies of the SMN2 backup gene, as…

Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families with support, reports that the Senate’s Health, Education, Labor and Pensions (HELP) Committee has passed seven bills as part of its “step-by-step” approach to Innovation and…

The Prudential Spirit of Community Awards, a nationwide program that honors young people for outstanding acts of volunteerism, has selected its top two Illinois youth volunteers of 2016, including Jungin Angie Lee, 16, of Naperville, who co-founded a nonprofit organization that generated nearly $200,000 over the past nine years with the…

New knowledge in stem cell research may help scientists advance cell therapies for motor neuron diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). Human neurons are as diverse as other cell types. Understanding how neurons diversify from stem cells would allow for more precise models of disease…

Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families with support, reports that it has been carefully monitoring U.S. Senate consideration of bill H.R. 6, the 21st Century Cures Act, on Capitol Hill. Congress describes…

A new research paper published in Annals of Clinical and Translational Neurology reveals promising biomarkers for disease progression assessment of spinal muscular atrophy (SMA) in infants, as well as monitoring of treatment effectiveness. The paper is titled “Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.” Early…

Motor neurons in spinal muscular atrophy (SMA) patients express fewer proteins associated with neuronal development than those in healthy individuals, according to a study that used induced pluripotent stem cells from individuals in both groups to create autologous motor neurons. The researchers — led by Heidi Fuller from The Robert Jones and…

Cure SMA has urged the U.S. Food and Drug Administration (FDA) to ensure that user fee agreement funds are used to incorporate and strengthen patient opinions in the drug development and approval processes. The plea came in Cure SMA testimony at a December meeting, one of several monthly meetings…

Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future — an initiative designed to expand the company’s drug pipeline and advance its muscle biology-directed drug candidates toward late-stage development and marketing strategies. The biopharmaceutical company specializes in first-in-class muscle activators for people with impaired muscle function, including spinal muscular atrophy (SMA) patients. The main…

Ionis Pharmaceuticals announced it has completed its target enrollment of the Phase 3 CHERISH study, a trial designed to support marketing approval of the drug candidate nusinersen in children with spinal muscular atrophy (SMA). The achievement earned Carlsbad, California-based Ionis a milestone payment of $2.15 million from Biogen, of Cambridge, Mass. According…