GAUCHER'S DISEASE TYPE I

Clinical trials for GAUCHER'S DISEASE TYPE I explained in plain language.

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Completed 1

New hope for kids with rare genetic disorder

Disease control Completed

This study tested a daily pill called eliglustat in children aged 2 to 18 with Gaucher disease, a rare genetic condition. It aimed to see if the drug was safe, how the body processed it, and if it could help control the disease by improving blood counts, organ size, and bone heal…

Matched conditions: GAUCHER'S DISEASE TYPE I

Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control

Last updated Mar 30, 2026 14:32 UTC

GAUCHER'S DISEASE TYPE I

Get alerted when new GAUCHER'S DISEASE TYPE I trials appear

New hope for kids with rare genetic disorder