Trials are taking place for the new antisense oligonucleotide drug by Biogen.

Similar to Spinraza, but believed to have a better stability in the human body. This means larger dose can be administered through spinal tap at longer intervals compared to Spinraza. We believe they are aiming for every 180 days but yet to be confirmed.

As this is a new drug the first stage will be to understand safety and how the drug actually behaves. So smaller doses will be given to start with and they will continue to increase it until they find a maximum safe dose.

This trial will be aimed at healthy people or those on gene therapy.

TreatSMA have contacted Biogen for more information.