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Emerging Concepts in Pathogenesis, Multiomics Applications, and Clinical Research in Lymphangioleiomyomatosis
Jane J Yu1, Nishant Gupta1, Minzhe Guo2
1Division of Pulmonary, Critical Care and Sleep Medicine, Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio.
Lymphangioleiomyomatosis (LAM) is a rare lung disease. Recent progress, including FDA-approved therapy and guidelines, offers hope for new biomarkers and treatments.
Area of Science:
- Pulmonary Medicine
- Oncology
- Rare Diseases
Background:
- Lymphangioleiomyomatosis (LAM) is a rare, female-predominant neoplasm.
- Characterized by abnormal smooth muscle-like cell infiltration in the lungs, leading to cystic changes and respiratory failure.
Purpose of the Study:
- To highlight recent advancements in Lymphangioleiomyomatosis (LAM) research and clinical care.
- To emphasize the collaborative efforts driving progress in rare lung diseases.
Main Methods:
- Review of recent developments in LAM research and clinical practice.
- Integration of bioinformatics and experimental approaches.
Main Results:
- Development of an FDA-approved therapy for LAM.
- Establishment of a diagnostic biomarker and a global clinical network.
- Creation of clinical practice guidelines for LAM management.
Conclusions:
- LAM serves as a model for rare lung disease progress.
- Ongoing research promises novel biomarkers and therapies for LAM in the near future.
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