Gaucher disease
Clinical trials for Gaucher disease explained in plain language.
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Japanese study checks safety of gaucher drug VPRIV in Real-World use
Disease control CompletedThis completed study in Japan looked at the safety and effectiveness of the drug VPRIV (velaglucerase alfa) in 63 people with Gaucher disease. Participants were either new to treatment or switching from another Gaucher therapy. The main goal was to track side effects and serious …
Matched conditions: GAUCHER DISEASE
Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 09:11 UTC
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Scanners peer into brains of gaucher and parkinson patients
Knowledge-focused CompletedThis completed study used PET and MRI scans to compare brain dopamine function in people with Gaucher disease or Parkinson disease, their family members, and healthy volunteers. Researchers aimed to see if certain genetic mutations cause early changes in dopamine storage. The stu…
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Sep 10, 2026 00:00 UTC
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Can brain scans and typing tests reveal early Parkinson's signs?
Knowledge-focused CompletedThis study uses advanced PET brain scans to measure dopamine, acetylcholine, and tau protein levels in people with GBA gene mutations, both with and without Parkinson's disease. Researchers also analyze typing patterns on a computer or touchscreen to detect subtle motor changes. …
Matched conditions: GAUCHER DISEASE
Sponsor: Pacific Parkinson's Research Centre • Aim: Knowledge-focused
Last updated Sep 04, 2026 00:00 UTC
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Blood test uncovers rare diseases in patients with mysterious enlarged spleen
Knowledge-focused CompletedThis study looked for two rare genetic diseases, Gaucher disease and acid sphingomyelinase deficiency (ASMD), in 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 enzyme activity …
Matched conditions: GAUCHER DISEASE
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC