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Alpelisib in PIK3CA-related overgrowth syndrome
The purpose of this study is to find out if alpelisib (BYL719; the “study drug”), is safe and effective (can help) people who have PIK3CA-related overgrowth spectrum (PROS) disease.
Related PI: Denise Adams, MD
Related Conditions: Complex Vascular Anomalies, Oncology
Topical Sirolimus for Microcystic Lymphatic Malformations
The purpose of this study is to see if a new topical (applied to the skin) formulation of rapamycin, PTX-022, is safe and effective in treating participants with microcystic Lymphatic Malformations (mLM).
Related PI: James R. Treat, MD; Denise Adams, MD
Related Conditions: Lymphatic Anomalies, Complex Vascular Anomalies, Complex Lymphatic Anomalies