Experiences with

Gangliosidosis

weekend is available to view: https://www.alextlc.org/digital-community-weekend-2021/ On Saturday, the Science Day, we have presentations about the new NHS Inherited White Matter Disorders Patient Registry and research updates on the following Leukodystrophies: - AMN & ALD - Alexander's Disease - GM1 gangliosidosis

Passage Bio are recruiting to two gene therapy trials: - Early onset and late onset infantile GM1 gangliosidosis Clinical trial information, including eligibility criteria: https://clinicaltrials.gov/ct2/show/NCT04713475 Register interest for participation in trial: https://www.pbgm01.com - Early