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GM1 gangliosidosis

Phase I/II gene transfer clinical trial for GM1 gangliosidosis delivering LYS-GM101

What is the purpose of this study?

The purpose of this trial will be to evaluate safety and efficacy of the delivery of LYS-GM101 as a treatment of GM1 gangliosidosis.

Trial at a glance

ConditionStudy typeClinical sites
GM1interventionalSite selection is ongoing

What next :

  • Patients will be eligible to participate from a variety of geographical locations.
  • We currently envisage that patients recruited to the trial will need to relocate to one of the clinical trial sites for an initial period of a few weeks. Additional visits to the clinical site, for a day or two, will be planned. 
  • Face-to-face meetings between families and the investigator at the clinical trial site, will take place before formal inclusion.
  • An information letter and informed consent document, in the parents’ own language, will be given to parents before inclusion in the study.
  • Arrangements for travel to clinical trial sites will be made through our partners. They will provide a safe and independent logistical resource to families of children recruited to the trial. This resource will continue to support families throughout the time their children are on the clinical trial.
  • We will set up a patient/family logistics and reimbursement policy to be implemented for patients recruited to the study.
  • If you are interested in participating and would like to contact someone, please email:


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