GANGLIOSIDOSES, GM2

Clinical trials for GANGLIOSIDOSES, GM2 explained in plain language.

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Race against time: new pill aims to slow devastating childhood brain disease

Disease control Recruiting now

This study is testing whether a daily oral medicine called nizubaglustat can slow the progression of two rare, severe genetic brain diseases (GM1 and GM2 gangliosidosis) in children. For 18 months, about 75 participants aged 4 and older will receive either the real medicine or a …

Matched conditions: GANGLIOSIDOSES, GM2

Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control

Last updated Mar 30, 2026 14:32 UTC

GANGLIOSIDOSES, GM2

Get alerted when new GANGLIOSIDOSES, GM2 trials appear

Race against time: new pill aims to slow devastating childhood brain disease