C9ORF72 Mice Point to Gain of Toxic Function in ALS, FTD Does C9ORF72 Repeat RNA Promote Protein Phase Transitions? RNA Regulator Locked Out of Nucleus by C9ORF72 Repeats Listen Up, Gene Silencing Strikes a Chord at RNA Meeting Stressed Out: RNA-binding P
Two drugs that boost α-synuclein clearance have passed Phase 1 safety benchmarks, and a stem cell strategy is poised to enter trials, reported scientists at SfN 2015.
Meet RBM45. This RNA-binding protein and relatively new player in the ALS field associates with stress-induced structures in the cytoplasm and nucleus.
In a process known as repeat-associated non-ATG translation, neurons and glia make alanine, serine, cysteine, and leucine chains from the huntingtin gene.
Both antibodies might be working, experts say, but the latest data released at the CTAD conference remain tantalizingly unclear. Trialists urgently need progression predictors and better outcome measures.
Gantenerumab, Aducanumab: Bobbing Up and Down While Navigating Currents of Trial Design Outcomes, Outcomes: Cognition is Crux of New Alzheimer’s Trials Truly New to Déjà Vu: Investigational Therapy News at CTAD Truly New to Déjà Vu: For Five Hopefuls, Lig
The CTAD conference featured discussion among many scientists of how to measure a drug’s effect in pre-dementia patients who are so mildly impaired that established tools have trouble picking up improvement. Better cognitive tools are needed.
Several therapeutic approaches in Phase 3 reported updates at the CTAD conference earlier this month in Barcelona. Read about levetiracetam, deep brain stimulation, and scyllo-inositol.