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Engineering Tendon Assembloids to Probe Cellular Crosstalk in Disease and Repair
Published on: March 22, 2024
Clinical trials and basic research: defining mechanisms and improving treatment in connective tissue disease
Lewis J Rubin1, Carol M Black, Christopher P Denton
1Division of Pulmonary and Critical Care Medicine, University of California San Diego, Campus Point Drive, La Jolla, California 92037-7381, USA. ljrubin@ucsd.edu
Abstract:
Despite advances in elucidating the pathogenic factors responsible for its development, systemic sclerosis remains complex and poorly understood, and treatment options are limited. Multidisciplinary collaborative efforts are needed to better characterize clinical and prognostic parameters and to design and implement large-scale clinical trials in well defined populations with therapies that target potential disease modulators.
Insights
Systemic sclerosis is complex with limited treatments. Further research requires multidisciplinary collaboration for large-scale trials targeting disease modulators.
Area of Science:
- Rheumatology
- Immunology
- Genetics
Background:
- Systemic sclerosis pathogenesis is incompletely understood despite advances.
- Current treatment options for systemic sclerosis are limited.
- Disease complexity necessitates novel therapeutic strategies.
Purpose of the Study:
- To highlight the need for multidisciplinary collaboration in systemic sclerosis research.
- To emphasize characterizing clinical and prognostic parameters.
- To advocate for large-scale clinical trials targeting disease modulators.
Main Methods:
- Literature review and expert consensus synthesis.
- Analysis of current research gaps in systemic sclerosis.
- Framework development for future clinical trial design.
Main Results:
- Identification of key areas for improved understanding of systemic sclerosis.
- Outlined strategies for better patient stratification in clinical trials.
- Emphasis on targeting specific disease modulators.
Conclusions:
- Multidisciplinary collaboration is crucial for advancing systemic sclerosis research.
- Standardized characterization of clinical and prognostic parameters is essential.
- Well-defined populations and targeted therapies are key for future trials.
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