HUNTER SYNDROME
Clinical trials for HUNTER SYNDROME explained in plain language.
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Hunter syndrome patients continue treatment in Long-Term safety extension trial
Disease control OngoingThis study is a follow-up for people with Hunter syndrome and cognitive problems who completed earlier studies. It aims to check the long-term safety of two medicines given together: idursulfase-IT (injected into the spine) and Elaprase (given through a vein). About 6 children an…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get donor cells to grow in the patient's body while keeping transplant-related deaths low. Participants receive chemotherapy drugs (busulfan and f…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 12, 2026 13:39 UTC
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Hunter syndrome immune study pulled before it began
Disease control TerminatedThis study aimed to see if a combination of immune-suppressing drugs could prevent the body from making antibodies that block the enzyme therapy ELAPRASE in boys with Hunter syndrome who had never been treated before. The plan was to give rituximab, methotrexate, and intravenous …
Matched conditions: HUNTER SYNDROME
Phase: PHASE4 • Sponsor: Takeda • Aim: Disease control
Last updated May 07, 2026 18:38 UTC