Lifeline offered for rare growth disorder patients

NCT ID NCT04085653

Summary

This program provides access to the drug alpelisib for patients with PIK3CA-Related Overgrowth Spectrum (PROS), a rare genetic condition causing abnormal tissue growth. It's designed for patients with severe or life-threatening cases who have no other feasible treatment options. The program allows doctors to use this medication while following specific safety guidelines and monitoring requirements.

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Contacts and locations

Study contacts

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Conditions

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