GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
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Japanese study confirms VPRIV safety for gaucher patients
Disease control CompletedThis study looked at how safe and effective VPRIV is for people with Gaucher disease in Japan. It included 63 patients who were either new to treatment or switching from another drug. Researchers tracked side effects and changes in blood health to see how well the medicine worked…
Matched conditions: GAUCHER DISEASE
Sponsor: Takeda • Aim: Disease control
Last updated May 17, 2026 04:12 UTC
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Simple blood test uncovers rare diseases behind mysterious enlarged spleens
Knowledge-focused CompletedThis study tested a simple blood spot test to find two rare genetic diseases (Gaucher and ASMD) in 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. The goal was to see how common these diseases are in this group. The test measure…
Matched conditions: GAUCHER DISEASE
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 17, 2026 04:28 UTC
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Scans reveal brain changes in gaucher and Parkinson's families
Knowledge-focused CompletedThis study used PET and MRI scans to compare brain dopamine function in people with Gaucher disease, Parkinson's disease, or a family history of these conditions, against healthy volunteers. Researchers aimed to detect early brain changes linked to glucocerebrosidase gene mutatio…
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 13, 2026 16:03 UTC