VON GIERKE'S DISEASE (GSD TYPE IA)

Clinical trials for VON GIERKE'S DISEASE (GSD TYPE IA) explained in plain language.

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Gene therapy patients monitored for years in rare disease study

Disease control Completed

This study followed 12 adults with a rare genetic liver disorder (GSDIa) for 4 to 6 years after they received a single dose of an experimental gene therapy. The main goal was to check for any long-term safety issues from the treatment. Researchers also monitored whether the thera…

Matched conditions: VON GIERKE'S DISEASE (GSD TYPE IA)

Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control

Last updated Mar 27, 2026 12:40 UTC

VON GIERKE'S DISEASE (GSD TYPE IA)

Get alerted when new VON GIERKE'S DISEASE (GSD TYPE IA) trials appear

Gene therapy patients monitored for years in rare disease study