Last-Resort drug access opens for rare MPS VII patients
NCT ID NCT03775174
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 33 times
Summary
This program provides expanded access to Mepsevii for people with MPS VII (Sly Syndrome) who have no other treatment options. Mepsevii is an enzyme replacement therapy designed to help manage the disease. Patients must meet specific eligibility criteria to be considered.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MUCOPOLYSACCHARIDOSIS VII are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.