Precision Medicine in Neurodegenerative Disorders, Part Two, Volume 193 discusses a major transformation fueled by the recognition that what has worked for developing symptomatic therapies doesn't necessarily work for developing disease modifying therapies. The former depends on addressing convergent, common denominators of disease, present in most of those affected. The latter on divergent biological signals, present in some but absent in most. This work encompasses two volumes separated into multiple sections, including conceptual frameworks, pitfalls in definitions, cohorts, and measures of progression, basic science development, and clinical trials and therapeutic approaches. Summarizes theory and research on precision medicine in neurodegenerative disorders Covers basic biology, clinical trials and therapeutics Includes disease mechanisms, genetic subtypes, and more INDICE: Part 3. Basic Science Development 1. Role of Rodent Models in Advancing Precision Medicine for Parkinson's disease 2. The Allure and Pitfalls of the Prion-like aggregation in neurodegeneration 3. The Shift to a Proteinopenia Paradigm in Neurodegeneration 4. Disease mechanisms as Subtypes: Lysosomal dysfunction in the Endolysosomal Parkinson's disease subtype 5. Disease mechanisms as Subtypes: Mitochondrial and bioenergetic dysfunction 6. Disease mechanisms as Subtypes: Immune dysfunction in Parkinson's disease 7. Disease mechanisms as Subtypes: Inflammation in Parkinson disease and related disorders 8. Disease mechanisms as Subtypes: Microbiome 9. LRRK2: Genetic mechanisms versus Genetic subtypes 10. Genetic mechanism vs. Genetic Subtypes: The example of GBA 11. Subtyping monogenic disorders: Huntington disease Part 4. Clinical Trials and therapeutic approaches 12. Disease-modifying versus symptomatic treatments: splitting over lumping 13. Restorative Cell and Gene Therapies for Parkinson Disease 14. The promise and challenges of extracellular vesicles in the diagnosis of neurodegenerative diseases 15. Therapeutic potential of extracellular vesicles in neurodegenerative disorders 16. Lessons from Anti-Amyloid-ß Immunotherapies in Alzheimer's disease 17. Lessons from immunotherapies in multiple sclerosis 18. Adaptive Clinical Trials and Master Protocols 19. Role of novel end-points and evaluations of response in Parkinson Disease 20. Leveraging the regulatory framework to facilitate drug development in Parkinson's disease
- ISBN: 978-0-323-85555-6
- Editorial: Elsevier
- Encuadernacion: Cartoné
- Páginas: 380
- Fecha Publicación: 01/05/2023
- Nº Volúmenes: 1
- Idioma: Inglés