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LYS-GM101 for GM1 Gangliosidosis
Study Summary
This trial is testing a gene therapy for a disease that causes damage to the nervous system. They will be testing how safe and effective it is.
- GM1 Gangliosidosis
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Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
Frequently Asked Questions
Are there any patient slots available for this research endeavor?
"According to the records on clinicaltrials.gov, this investigation is no longer seeking new participants as recruitment ended in 8/30/2022 since first posting on 5/11/2021. However, there are still 7 other medical trials that are currently calling for volunteers."
What is the primary goal of this research endeavor?
"The primary outcome measure for this trial will be observed over a 6-month period, and focuses on the incidence of any adverse events or serious health risks. Secondary measurements include developmental changes (as gauged by VABS-II, BSID-III or KABC-II), plus blood and cerebrospinal fluid biomarkers such as beta galactosidase activity levels."
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