GM1 GANGLIOSIDOSIS, TYPE I

Clinical trials for GM1 GANGLIOSIDOSIS, TYPE I explained in plain language.

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Doctors test First-Ever prenatal gene therapy for devastating baby disease

Disease control Not yet recruiting

This is a first-in-human safety study testing a gene therapy given to babies before they are born. It is for fetuses diagnosed with GM1 gangliosidosis, a rare and severe genetic disorder that damages the brain and body. Doctors will inject a modified virus carrying a healthy gene…

Matched conditions: GM1 GANGLIOSIDOSIS, TYPE I

Phase: PHASE1 • Sponsor: Tippi Mackenzie • Aim: Disease control

Last updated Mar 23, 2026 15:18 UTC

GM1 GANGLIOSIDOSIS, TYPE I

Get alerted when new GM1 GANGLIOSIDOSIS, TYPE I trials appear

Doctors test First-Ever prenatal gene therapy for devastating baby disease