MUCOPOLYSACCHARIDOSIS VII
Clinical trials for MUCOPOLYSACCHARIDOSIS VII explained in plain language.
Never miss a new study
Get alerted when new MUCOPOLYSACCHARIDOSIS VII trials appear
Sign up with your email to follow new studies for MUCOPOLYSACCHARIDOSIS VII, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Last-Resort drug access opens for rare sly syndrome patients
Disease control AVAILABLEThis 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.
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
-
Rare disease study tracks MPS VII over time
Knowledge-focused Recruiting nowThis study monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular che…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC
-
New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab re…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
-
Massive french study aims to unlock secrets of rare MPS diseases
Knowledge-focused Recruiting nowThis observational study will follow up to 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will collect medical data from patient records and ongoing checkups to map how the diseases progress and how current treatments affec…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC