Ahmad Kilo,  —

RaNA Therapeutics recently released preclinical data on its gene upregulation technology, which selectively triggers gene expression within cells, as a treatment for spinal muscular atrophy (SMA). The data were given in a poster presentation at the recent Keystone Symposium for Chromatin and Epigenetics in British Columbia, Canada. SMA, a major cause of infant mortality, results from mutations or deletions of SMN1 gene…

Cure SMA recently published a 2015 review of its advocacy work, highlighting the programs it provides to families living with Spinal Muscular Atrophy (SMA) that aid in caring for those with SMA and offering the emotional support of a unified community who understands the struggles of the disease. In its efforts to support the continued…

A team of researchers from Harvard University and the Karolinska Institute in Sweden unveiled the cellular pathway leading to the specific death of motor neurons in spinal muscular atrophy (SMA). The study entitled “Genome-wide RNA-Seq of Human Motor Neurons Implicates Selective ER Stress Activation in Spinal Muscular…

Albany Molecular Research (AMRI), a contract research and manufacturing organization that provides drug discovery, development, cGMP manufacturing and aseptic fill and finish to the pharmaceutical and biotechnology industries, recently announced it has been granted a 10-year federal contract award care of the National Institutes of Health (NIH) to secure their drug development…

Swiss global health-care company Roche announced just last week that it has purchased Trophos, a privately-owned Marseille, France-based clinical stage pharmaceutical company that specializes in mitochondrial targeted compounds that work to improve the function and survival of stressed cells by preventing mitochondrial permeability transition, hence helping prevent cell death. Roche has taken an interest in…

Dallas-based biotechnology company AveXis, Inc. announced yesterday that they have successfully dosed the first patient in the intermediate cohort of their Gene Transfer Clinical Trial for chariSM A™ for Spinal Muscular Atrophy Type 1. The news comes just days after the company announced on December 5th that the dosing phase in the low-dose…

Early in September, the story of a 4-year-old girl from West Monroe, with spinal muscular atrophy (SMA), Ella Ecker, aired on Fox News about her one big wish to go to Disney World. Soon, donations for Ella’s wish to come true began coming in, but what sealed…

While SMA is a rare disease that many people are unaware of, an active network of advocacy groups work worldwide to raise funds and awareness about the disease. As part of this effort, the Angel Gala in Canada will continue its legacy of supporting spinal muscular atrophy (SMA)…

Lydia Peters cutting the ribbon during the playground’s opening ceremony. Finally, parents of children with special needs can breathe easier when their little ones ask for an afternoon at the playground. It was last August 22, when over a hundred locals and public figures gathered to witness the grand…