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…
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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…
Roche recently announced that a new investigational medicine for the treatment of spinal muscular atrophy (SMA) — RG7916 — will soon advance to its first clinical trial. The study will provide information on the safety and tolerability, and investigate the pharmacokinetics (absorption, distribution, metabolism and excretion) of RG7916 in healthy individuals. In April 2015, the Moonfish clinical…
Ionic Pharmaceuticals announced a landmark payment of $5 million from Biogen for the validation of an unnamed target to treat patients with an as-yet unrevealed neurological disorder. Ionis and Biogen have an extensive strategic partnership to develop drugs for neurological disorders; Ionis will continue to evaluate the newest target to…
Researchers have developed a novel method for detecting carriers of spinal muscular atrophy (SMA) using next generation sequencing screening. The new technique can easily be incorporated into current next generation sequencing screening panels, analyzing hundreds of genes involved in severe childhood diseases. While a rare disease, SMA is still the…
