Mutation in the Parkinson’s Gene DJ-1 Derails Splicing
Restoring proper gene editing assuaged mitochondrial defects in patient-derived neurons and organoids. Splicing errors may underlie other PD cases as well.
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At AAIC, Encouraging Safety Data on a Variety of Small-Molecule Candidates
Three BACE inhibitors, a γ-secretase modulator, and a phosphodiesterase inhibitor appeared safe in Phase 1 trials.
11th ICFTD Meeting in Sydney Sorts Out Clinical Subtypes
Can genetics please parse the confusing spectrum of frontotemporal dementias? Whole genome sequences make a start.
Colombian Cohort Delivers Data on Blood NfL
In 2,144 Colombian ADAD family members, plasma NfL in gene carriers rises as early as two decades before their symptoms start.
Blood Test Granted Breakthrough Status, To Be Tested in Trial
The FDA has prioritized review of C2N’s blood test for amyloid-β. A pivotal clinical trial will correlate the test with amyloid PET scans.
Blood Tests for Amyloid Step Out at CTAD
With plasma tests performing in AIBL staging, scientists are sharing data across platforms and cohorts, and tackling standardization to avoid time lost to irreproducibility.
Is There No End to Tau’s Toxic Tricks?
At SfN 2017, some of the lesser-known tau toxicities came in for deep scrutiny.
Will Next-Gen BACE Inhibitors Dodge Side Effects?
Despite no warning signs in ongoing clinical trials, researchers are searching for safer drugs, and better biomarkers to measure what they do.
FDA Advisors Will Take up Aducanumab on November 6
The day-long advisory committee meeting will be broadcast live online. Prerecorded presentations are to be available November 4; the public can submit comments.
Virtual Meetings Try Out Different Formats
A recent genetics symposium drew positive reviews for its approach of following up prerecorded, on-demand talks with a live Q&A session.
Parkinson’s Treatments Go After Genetic Targets
Researchers at AAT-AD/PD discussed investigational PD treatments that aim to modify disease by hitting genetic risk factors.
Antibody Against α-Synuclein Looks Safe In Phase 1
Treatments targeting the main pathological protein of Parkinson’s disease are moving toward the clinic, with two immunotherapies passing Phase 1 safety benchmarks.
Parsing How Alzheimer’s Genetic Risk Works Through Microglia
Scientists at AD/PD 2019 see a Goldilocks of microglial activation: Both too little and too much is bad in an injured brain. How could a therapy make it just right?
No Skin Off Your Nose: New Way to Diagnosing Parkinson’s?
Two papers report that skin samples from people with Parkinson’s disease contain α-synuclein seeds that can be robustly amplified, paving the way for a reliable test for the disease.
Curbing Fatty Acids Means No Parkinson’s—If You Are a Mouse
Reducing levels of monounsaturated fatty acids lowered α-synuclein toxicity and prevented movement symptoms in mice. Scientists say the data boost the α-synuclein tetramer hypothesis.
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