GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
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Gaucher drug VPRIV under the microscope: safety check in 63 patients
Disease control CompletedThis study followed 63 people with Gaucher disease in Japan who were either starting VPRIV for the first time or switching from another treatment. Researchers tracked side effects and checked if the drug helped improve key health measures like hemoglobin levels. The goal was to c…
Matched conditions: GAUCHER DISEASE
Sponsor: Takeda • Aim: Disease control
Last updated May 22, 2026 13:53 UTC
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Simple blood test uncovers rare diseases in patients with unexplained enlarged spleen
Knowledge-focused CompletedThis study looked at 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. Researchers used a simple blood spot test to check for two rare genetic disorders: Gaucher disease and acid sphingomyelinase deficiency (ASMD). The goal was to…
Matched conditions: GAUCHER DISEASE
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 26, 2026 09:31 UTC
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Scans reveal brain clues in Parkinson's and gaucher patients
Knowledge-focused CompletedThis study used PET and MRI scans to compare how the brain uses dopamine in people with Gaucher disease or Parkinson's, their family members, and healthy volunteers. The goal was to find early brain changes linked to a specific gene mutation. About 64 adults aged 21 and older too…
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC