HUNTER SYNDROME
Clinical trials for HUNTER SYNDROME explained in plain language.
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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 the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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Hunter syndrome study pulled before it even started
Disease control TerminatedThis study aimed to see if giving a combination of immune-suppressing drugs (rituximab, methotrexate, and IVIG) alongside the standard enzyme therapy ELAPRASE could prevent patients with Hunter syndrome from developing harmful antibodies. It was designed for boys who had never re…
Matched conditions: HUNTER SYNDROME
Phase: PHASE4 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Hunter syndrome drug safety tracked in extended trial
Disease control OngoingThis study is a follow-up for people with Hunter syndrome and cognitive problems who already took part in earlier studies. It aims to check the long-term safety of the drug idursulfase-IT given with Elaprase. About 6 children and adults will continue their same treatment to monit…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Home infusions may help patients stick to treatment
Knowledge-focused OngoingThis study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…
Matched conditions: HUNTER SYNDROME
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC