RaNA Therapeutics recently released preclinical data on its gene upregulation technology, which selectively triggers gene expression within cells, as a treatment for spinal…
Cure SMA recently published a 2015 review of its advocacy work, highlighting the programs it provides to families living with Spinal Muscular…
A team of researchers from Harvard University and the Karolinska Institute in Sweden unveiled the cellular pathway leading to the…
Albany Molecular Research (AMRI), a contract research and manufacturing organization that provides drug discovery, development, cGMP manufacturing and aseptic fill…
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…
Dallas-based biotechnology company AveXis, Inc. announced yesterday that they have successfully dosed the first patient in the intermediate cohort of their Gene…
Early in September, the story of a 4-year-old girl from West Monroe, with spinal muscular atrophy (SMA),…
While SMA is a rare disease that many people are unaware of, an active network of advocacy…
Lydia Peters cutting the ribbon during the playground’s opening ceremony. Finally, parents of children with special needs can breathe…
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