Gene Therapy trial now enrolling children ages 2-12 with type II GM1 gangliosidosis. The study is to determine if a gene therapy can help the body to make an enzyme. Read details at ClinicalTrials.gov
Juvenile GM1 Trial Open
- Details
Gene Therapy trial now enrolling children ages 2-12 with type II GM1 gangliosidosis. The study is to determine if a gene therapy can help the body to make an enzyme. Read details at ClinicalTrials.gov