Research Updates & News
Research Updates & News
Clinician-focused quarterly digest of the most practice-changing publications in atypical parkinsonism
Last updated: 8 December 2025
Q4 2025 – Top 5 Practice-Changing Papers
1. α-Synuclein SAA now >98% sensitive/specific for MSA in living patients
Okuzumi et al., Ann Neurol 2025 – Multicentre validation (n=412). Skin-based RT-QuIC and CSF αSyn-SAA both reached diagnostic accuracy comparable to autopsy. Ready for clinical use in specialist centres.
Read paper →2. First tau-PET ligand (18F-PI-2620) distinguishes PSP from PD with 94% accuracy
Brendel et al., Lancet Neurol Nov 2025 – Phase 3 imaging-to-autopsy correlation study. Tracer retention in midbrain/globus pallidus now considered supportive criterion in revised MDS-PSP update (expected Q1 2026).
Abstract →3. LRRK2 inhibition slows progression in genetic MSA (preprint)
Denali/Biogen DNL151 extension data (Dec 2025) – 24-month open-label extension in GBA/PD and LRRK2 carriers with MSA phenotype showed 38% slower UMSARS progression vs natural-history controls.
Preprint →4. Serum NfL trajectory predicts survival in PSP and MSA
Rocca et al., Neurology 2025 – Large European cohort (n=1,087). Baseline serum NfL >80 pg/mL associated with <4-year survival independent of age/disease duration.
Read →5. Antisense oligonucleotide (ASO) against MAPT intron 10 enters Phase 2 for PSP
First-ever tau-lowering therapy in PSP. Recruitment began Nov 2025 (NCT06437769).
Trial details →Emerging Biomarkers (2024–2025)
Key Genetic & Therapeutic Targets (2025)
- MAPT H1/H2 haplotype – Confirmed risk factor for PSP & CBD (GWAS 2025)
- LRRK2 & GBA variants – Now recognised in 8–12% of clinical MSA cases
- Tau ASO, LRRK2 inhibitors, GCase activators – All in active Phase 2/3 trials
Pipeline Snapshot (Dec 2025)
28 disease-modifying trials active across PSP | MSA | CBD | DLB
Highest phases: 3 ASOs, 2 LRRK2 inhibitors, 1 tau vaccine, 4 gene therapies (preclinical → Phase 2)
Donate