LYSOSOMAL DISEASES

Clinical trials for LYSOSOMAL DISEASES explained in plain language.

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Trials to join now! 1

Experimental gene therapy offers hope for children with fatal GM1 disease

Disease control Recruiting now

This study tests a one-time gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1…

Matched conditions: LYSOSOMAL DISEASES

Phase: PHASE1, PHASE2 • Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control

Last updated May 26, 2026 07:27 UTC

LYSOSOMAL DISEASES

Get alerted when new LYSOSOMAL DISEASES trials appear

Experimental gene therapy offers hope for children with fatal GM1 disease