Families of SMA, USA
Illinois, USA
Das FSMA / Aurora-Projekt
In March 2000, FSMA entered into a $2.6 million collaboration agreement with Aurora
Biosciences Corporation (Aurora) to screen over 500,000 compounds on 2 separate
mechanism-based assays (a promoter assay and a splicing assay) for use in high-
throughput screening for drug leads. The strategy behind both of the high-throughput
compound screens was to identify agents that increase the total amount of functional
SMN messenger RNA (mRNA) and protein in motor neuron cells. Approximately 1.16
unique compounds have been tested using the 2 assays. This effort has resulted in
identification of 16 compounds with confirmed biological activity.