Glycoproteinosis
MONDO:001773114 clinical trials for this condition and its sub-types.
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Broader categories
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First gene therapy trial launches for rare childhood disease MLIV
Disease control By invitation onlyThis early-stage trial tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV (MLIV), a rare genetic disorder. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety, but researchers will also look for any si…
Phase 1 • Sponsor: The Children's Hospital of Zhejiang University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:03 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 the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Phase 2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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Cervical cancer screening showdown: pap vs. blood test study pulled
Diagnosis CancelledThis study was designed to compare the standard Pap smear with a new blood test that looks for three proteins linked to cervical cell changes. It planned to include adults aged 18-85 in good health. However, the study was withdrawn before enrolling any participants, so no results…
Sponsor: Timser SAPI de CV • Aim: Diagnosis
Last updated Jun 27, 2026 09:09 UTC
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New study tracks rare disease MLIV to map its natural course
Knowledge-focused OngoingThis study looks back at medical records of 50 people with Mucolipidosis Type IV (MLIV) to learn how the disease typically progresses. Researchers want to know when children reach or lose developmental milestones, how their movement and vision change, and what lab results look li…
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 UTC