Last-Resort drug access opens for rare MPS VII patients
NCT ID NCT03775174
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 30 times
Summary
This program provides expanded access to Mepsevii for people with MPS VII (Sly syndrome) who have no other treatment options. It is designed for individual patients who meet specific criteria. The goal is to offer a potential treatment when no alternatives exist.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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