ALS is a complex disorder which the cause(s) are not entirely understood. However, scientists at the ALS Therapy Development Institute have discovered a number of immune system related aspects of the disease for which they are screening potential treatments, including this anti-sense oligoneuclueotide (ASO).
ASOs are relatively new therapeutic strategies and delivery mechanisms. Early stage PK/PD experiments will be crucial to determine whether or not to continue into large scale efficacy studies.
Topics in the Pipeline
Register now for our upcoming webinar on May 10th at 6pm: FDA approves Radicava - What do we need to know?
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November 28 is