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Author Spotlight: Insight Into Advances in Prion Diseases Research
Published on: August 11, 2023
847
Therapeutic strategies for prion disease: a practical perspective
Brian S Appleby1, Allyson Connor2, Han Wang2
1National Prion Disease Pathology Surveillance Center, Case Western Reserve University School of Medicine, 2085 Adelbert Rd, Rm 419, Cleveland, OH 44106, USA; Department of Neurology, University Hospitals Cleveland Medical Center, 3619 Park East Drive, Suite 206, Beachwood, OH 44122, USA.
Current Opinion in Pharmacology
|December 4, 2018
Summary
Conducting clinical trials for human prion diseases presents unique challenges due to their rarity and rapid progression. This review highlights key considerations for improving future trial design and patient enrollment in prion disease research.
Area of Science:
- Neurology
- Neurodegenerative Diseases
- Clinical Trial Design
Background:
- Human prion diseases are fatal, rapidly progressing neurodegenerative disorders.
- Currently, no effective treatments exist for human prion diseases.
- Past clinical trials, though unsuccessful, offer valuable insights for future research.
Purpose of the Study:
- To review the challenges in conducting clinical trials for prion diseases.
- To suggest practical considerations for improving future prion disease clinical trials.
Main Methods:
- Literature review of challenges in prion disease clinical trials.
- Analysis of factors affecting study enrollment and treatment effect measurement.
Main Results:
- Prion disease rarity, rapid progression, and clinical heterogeneity complicate study enrollment and outcome assessment.
- Study methodology significantly impacts both trial results and participant recruitment.
Conclusions:
- Addressing the unique aspects of prion diseases is crucial for effective clinical trial design.
- Practical considerations are needed to overcome recruitment and measurement challenges in prion disease research.
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