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Related Concept Videos

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Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
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Related Experiment Video

Updated: Jan 10, 2026

A Method to Study α-Synuclein Toxicity and Aggregation Using a Humanized Yeast Model
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Small molecule inhibitors targeting alpha-synuclein aggregation: Progress and future outlook.

Ishfaq Bashir Hajam1, Ishfaq Ahmad Ahanger1, Umar Rasool1

  • 1Department of Clinical Biochemistry, University of Kashmir, Srinagar, Jammu and Kashmir, India.

Advances in Protein Chemistry and Structural Biology
|November 27, 2025
PubMed
Summary

Small molecule inhibitors targeting alpha-synuclein (α-syn) aggregation show promise for Parkinson's disease (PD) treatment. This review explores their development, clinical trials, and future potential for disease modification.

Keywords:
Intrinsically disorder proteinNeurodegenerative diseasesParkinson’s diseaseProtein foldingSmall molecular probes

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Area of Science:

  • Neuroscience
  • Pharmacology
  • Biochemistry

Background:

  • Parkinson's disease (PD) is characterized by alpha-synuclein (α-syn) aggregation, causing neuronal dysfunction.
  • Targeting α-syn aggregation is a key therapeutic strategy for PD.

Purpose of the Study:

  • To review the progress in developing small molecule inhibitors for α-syn aggregation.
  • To update on the clinical trial status of these inhibitors and discuss emerging strategies.

Main Methods:

  • Literature review of small molecule inhibitors targeting α-syn aggregation.
  • Analysis of mechanisms of α-syn misfolding and aggregation.
  • Overview of clinical trial data and emerging therapeutic approaches.

Main Results:

  • Several small molecule inhibitors are in development to prevent or reverse α-syn aggregation.
  • Clinical trials are ongoing, with some showing promising results.
  • Emerging strategies like combination therapies and chaperone therapeutics may enhance efficacy.

Conclusions:

  • Small molecule inhibitors represent a promising avenue for disease-modifying treatments in PD.
  • Further research and clinical trials are essential to realize their full therapeutic potential.
  • Combination therapies and novel approaches could improve treatment outcomes for Parkinson's disease.