Espay A. Precision Medicine in Neurodegenerative Disorders. Part II 2023

Textbook in PDF format

Foreword
In Memoriam–Michael Parkinson
Preface
Contributors
Basic science development
Role of rodent models in advancing precision medicine for Parkinson's disease
The allure and pitfalls of the prion-like aggregation in neurodegeneration
The shift to a proteinopenia paradigm in neurodegeneration
Disease mechanisms as subtypes: Lysosomal dysfunction in the endolysosomal Parkinson's disease subtype
Disease mechanisms as Subtypes: Mitochondrial and bioenergetic dysfunction
Disease mechanisms as subtypes: Immune dysfunction in Parkinson's disease
Disease mechanisms as subtypes: Inflammation in Parkinson disease and related disorders
Disease mechanisms as subtypes: Microbiome
LRRK2: Genetic mechanisms vs genetic subtypes
Genetic mechanism vs genetic subtypes: The example of GBA
Subtyping monogenic disorders: Huntington disease
Clinical trials and therapeutic approaches
Disease-modifying vs symptomatic treatments: Splitting over lumping
Restorative cell and gene therapies for Parkinson's disease
The promise and challenges of extracellular vesicles in the diagnosis of neurodegenerative diseases
Therapeutic potential of extracellular vesicles in neurodegenerative disorders
Lessons from antiamyloid-β immunotherapies in Alzheimer's disease
Lessons from immunotherapies in multiple sclerosis
Adaptive clinical trials and master protocols
Role of novel endpoints and evaluations of response in Parkinson disease
Leveraging the regulatory framework to facilitate drug development in Parkinson's disease
Index