Last-Resort drug access opens for rare sly syndrome patients
NCT ID NCT03775174
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 2 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 on a case-by-case basis. The goal is to offer a potential treatment when no alternatives exist.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
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