Progress reports on six treatment options for spinal muscular atrophy (SMA), all currently in clinical trials, were spotlighted at the recent 2016 Annual SMA Conference in Anaheim, CA. The updates, provided by study representatives, shows that efforts are advancing toward new therapies for SMA. Four of the six treatment programs — AVXS-101, nusinersen, RG7800/RG7916, and…
Researchers at the National Tsing Hua University in Taiwan discovered seven new genes affecting severity and time of onset of spinal muscular atrophy (SMA). The finding offers researchers clues for what molecules to target in new drug development attempts. The study, “An Integrative Transcriptomic Analysis for Identifying…
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…
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 SMAÂ 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…
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…
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…
