Will RNA molecules that bump up or tamp down gene expression live up to their potential? Results from RNA-targeting therapies presented at the 70th annual meeting of the American Academy of Neurology in Los Angeles suggest children with spinocerebellar ataxia continue to improve years after starting approved treatments. However, prices are high and so far, health gains are modest. Will similar approaches for Huntington’s disease, ALS, familial amyloid polyneuropathy, and tauopathies fare better?
At AAN, the latest data from Phase 3 trials and open-label extensions of RNA-targeting therapies reveal a bit of benefit, but no cure.
Antisense oligonucleotides are raising hopes for tackling a wide range of brain disorders—is the enthusiasm warranted?
Biogen showed results from clinical trials of anti-Aβ and anti-α-synuclein antibodies at the American Academy of Neurology annual meeting.